KR102785448B1 - 신경퇴행성 질병을 위한 치료제 - Google Patents
신경퇴행성 질병을 위한 치료제 Download PDFInfo
- Publication number
- KR102785448B1 KR102785448B1 KR1020247014635A KR20247014635A KR102785448B1 KR 102785448 B1 KR102785448 B1 KR 102785448B1 KR 1020247014635 A KR1020247014635 A KR 1020247014635A KR 20247014635 A KR20247014635 A KR 20247014635A KR 102785448 B1 KR102785448 B1 KR 102785448B1
- Authority
- KR
- South Korea
- Prior art keywords
- disease
- patient
- leucine
- acetyl
- pharmaceutical composition
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Active
Links
- 230000004770 neurodegeneration Effects 0.000 title claims abstract description 236
- 208000015122 neurodegenerative disease Diseases 0.000 title claims abstract description 232
- 239000003814 drug Substances 0.000 title description 31
- 229940124597 therapeutic agent Drugs 0.000 title 1
- WXNXCEHXYPACJF-ZETCQYMHSA-N N-acetyl-L-leucine Chemical compound CC(C)C[C@@H](C(O)=O)NC(C)=O WXNXCEHXYPACJF-ZETCQYMHSA-N 0.000 claims abstract description 143
- 229960000669 acetylleucine Drugs 0.000 claims abstract description 142
- 150000003839 salts Chemical class 0.000 claims abstract description 65
- WXNXCEHXYPACJF-UHFFFAOYSA-N N-acetyl-leucine Chemical group CC(C)CC(C(O)=O)NC(C)=O WXNXCEHXYPACJF-UHFFFAOYSA-N 0.000 claims description 90
- 239000008194 pharmaceutical composition Substances 0.000 claims description 50
- 208000018737 Parkinson disease Diseases 0.000 claims description 27
- 239000003150 biochemical marker Substances 0.000 claims description 27
- 208000027089 Parkinsonian disease Diseases 0.000 claims description 21
- 206010034010 Parkinsonism Diseases 0.000 claims description 20
- 201000006417 multiple sclerosis Diseases 0.000 claims description 14
- 230000001149 cognitive effect Effects 0.000 claims description 12
- IVWWFWFVSWOTLP-YVZVNANGSA-N (3'as,4r,7'as)-2,2,2',2'-tetramethylspiro[1,3-dioxolane-4,6'-4,7a-dihydro-3ah-[1,3]dioxolo[4,5-c]pyran]-7'-one Chemical compound C([C@@H]1OC(O[C@@H]1C1=O)(C)C)O[C@]21COC(C)(C)O2 IVWWFWFVSWOTLP-YVZVNANGSA-N 0.000 claims description 9
- 102000006441 Dopamine Plasma Membrane Transport Proteins Human genes 0.000 claims description 5
- 108010044266 Dopamine Plasma Membrane Transport Proteins Proteins 0.000 claims description 5
- 238000005259 measurement Methods 0.000 claims description 5
- 238000002600 positron emission tomography Methods 0.000 claims 4
- 238000002603 single-photon emission computed tomography Methods 0.000 claims 4
- 230000001939 inductive effect Effects 0.000 claims 3
- 238000011282 treatment Methods 0.000 description 199
- 208000024891 symptom Diseases 0.000 description 143
- 230000005021 gait Effects 0.000 description 102
- 206010008025 Cerebellar ataxia Diseases 0.000 description 101
- 238000012360 testing method Methods 0.000 description 93
- 241000699670 Mus sp. Species 0.000 description 85
- 102000014461 Ataxins Human genes 0.000 description 83
- 108010078286 Ataxins Proteins 0.000 description 83
- 208000009415 Spinocerebellar Ataxias Diseases 0.000 description 83
- 201000004562 autosomal dominant cerebellar ataxia Diseases 0.000 description 83
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 66
- 201000010099 disease Diseases 0.000 description 60
- 206010003591 Ataxia Diseases 0.000 description 56
- 230000006872 improvement Effects 0.000 description 56
- 238000004458 analytical method Methods 0.000 description 54
- 230000007423 decrease Effects 0.000 description 54
- 238000000034 method Methods 0.000 description 51
- 230000007659 motor function Effects 0.000 description 47
- 230000002132 lysosomal effect Effects 0.000 description 36
- 206010061818 Disease progression Diseases 0.000 description 32
- 230000005750 disease progression Effects 0.000 description 32
- 230000033001 locomotion Effects 0.000 description 31
- 206010029864 nystagmus Diseases 0.000 description 30
- 208000011580 syndromic disease Diseases 0.000 description 30
- 229940079593 drug Drugs 0.000 description 28
- 230000000750 progressive effect Effects 0.000 description 27
- 208000015439 Lysosomal storage disease Diseases 0.000 description 26
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 description 25
- 230000000694 effects Effects 0.000 description 25
- 230000006870 function Effects 0.000 description 25
- 230000003247 decreasing effect Effects 0.000 description 23
- 230000000007 visual effect Effects 0.000 description 21
- 201000011240 Frontotemporal dementia Diseases 0.000 description 20
- 208000012886 Vertigo Diseases 0.000 description 20
- 239000003826 tablet Substances 0.000 description 20
- 231100000889 vertigo Toxicity 0.000 description 20
- 102100022548 Beta-hexosaminidase subunit alpha Human genes 0.000 description 19
- 210000004556 brain Anatomy 0.000 description 19
- 230000006735 deficit Effects 0.000 description 19
- 208000008955 Mucolipidoses Diseases 0.000 description 18
- 208000001089 Multiple system atrophy Diseases 0.000 description 18
- 239000000203 mixture Substances 0.000 description 18
- 208000021811 Sandhoff disease Diseases 0.000 description 17
- 230000004064 dysfunction Effects 0.000 description 17
- 210000002950 fibroblast Anatomy 0.000 description 17
- 208000022292 Tay-Sachs disease Diseases 0.000 description 16
- 210000003141 lower extremity Anatomy 0.000 description 16
- 230000004112 neuroprotection Effects 0.000 description 16
- 230000002441 reversible effect Effects 0.000 description 16
- 208000024827 Alzheimer disease Diseases 0.000 description 15
- 241000699666 Mus <mouse, genus> Species 0.000 description 15
- 208000002740 Muscle Rigidity Diseases 0.000 description 15
- 230000001144 postural effect Effects 0.000 description 15
- 201000002212 progressive supranuclear palsy Diseases 0.000 description 15
- 206010044565 Tremor Diseases 0.000 description 14
- 230000035508 accumulation Effects 0.000 description 14
- 238000009825 accumulation Methods 0.000 description 14
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 description 14
- 150000002339 glycosphingolipids Chemical class 0.000 description 14
- 230000004434 saccadic eye movement Effects 0.000 description 14
- 208000014060 Niemann-Pick disease Diseases 0.000 description 13
- 230000006866 deterioration Effects 0.000 description 13
- 230000001771 impaired effect Effects 0.000 description 13
- 230000008449 language Effects 0.000 description 13
- 239000007788 liquid Substances 0.000 description 13
- 208000005340 mucopolysaccharidosis III Diseases 0.000 description 13
- 230000000384 rearing effect Effects 0.000 description 13
- 230000008901 benefit Effects 0.000 description 12
- 230000002490 cerebral effect Effects 0.000 description 12
- 230000007547 defect Effects 0.000 description 12
- 230000002068 genetic effect Effects 0.000 description 12
- 230000002829 reductive effect Effects 0.000 description 12
- 206010013887 Dysarthria Diseases 0.000 description 11
- 206010017577 Gait disturbance Diseases 0.000 description 11
- 238000003745 diagnosis Methods 0.000 description 11
- 238000010172 mouse model Methods 0.000 description 11
- 101710137189 Amyloid-beta A4 protein Proteins 0.000 description 10
- 101710151993 Amyloid-beta precursor protein Proteins 0.000 description 10
- 102100022704 Amyloid-beta precursor protein Human genes 0.000 description 10
- 239000013543 active substance Substances 0.000 description 10
- DZHSAHHDTRWUTF-SIQRNXPUSA-N amyloid-beta polypeptide 42 Chemical compound C([C@@H](C(=O)N[C@@H](C)C(=O)N[C@@H](CCC(O)=O)C(=O)N[C@@H](CC(O)=O)C(=O)N[C@H](C(=O)NCC(=O)N[C@@H](CO)C(=O)N[C@@H](CC(N)=O)C(=O)N[C@@H](CCCCN)C(=O)NCC(=O)N[C@@H](C)C(=O)N[C@H](C(=O)N[C@@H]([C@@H](C)CC)C(=O)NCC(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](CCSC)C(=O)N[C@@H](C(C)C)C(=O)NCC(=O)NCC(=O)N[C@@H](C(C)C)C(=O)N[C@@H](C(C)C)C(=O)N[C@@H]([C@@H](C)CC)C(=O)N[C@@H](C)C(O)=O)[C@@H](C)CC)C(C)C)NC(=O)[C@H](CC=1C=CC=CC=1)NC(=O)[C@@H](NC(=O)[C@H](CC(C)C)NC(=O)[C@H](CCCCN)NC(=O)[C@H](CCC(N)=O)NC(=O)[C@H](CC=1N=CNC=1)NC(=O)[C@H](CC=1N=CNC=1)NC(=O)[C@@H](NC(=O)[C@H](CCC(O)=O)NC(=O)[C@H](CC=1C=CC(O)=CC=1)NC(=O)CNC(=O)[C@H](CO)NC(=O)[C@H](CC(O)=O)NC(=O)[C@H](CC=1N=CNC=1)NC(=O)[C@H](CCCNC(N)=N)NC(=O)[C@H](CC=1C=CC=CC=1)NC(=O)[C@H](CCC(O)=O)NC(=O)[C@H](C)NC(=O)[C@@H](N)CC(O)=O)C(C)C)C(C)C)C1=CC=CC=C1 DZHSAHHDTRWUTF-SIQRNXPUSA-N 0.000 description 10
- 210000001508 eye Anatomy 0.000 description 10
- 239000003550 marker Substances 0.000 description 10
- 230000037230 mobility Effects 0.000 description 10
- 238000010176 18-FDG-positron emission tomography Methods 0.000 description 9
- ZCXUVYAZINUVJD-AHXZWLDOSA-N 2-deoxy-2-((18)F)fluoro-alpha-D-glucose Chemical compound OC[C@H]1O[C@H](O)[C@H]([18F])[C@@H](O)[C@@H]1O ZCXUVYAZINUVJD-AHXZWLDOSA-N 0.000 description 9
- 208000011990 Corticobasal Degeneration Diseases 0.000 description 9
- WTDRDQBEARUVNC-LURJTMIESA-N L-DOPA Chemical compound OC(=O)[C@@H](N)CC1=CC=C(O)C(O)=C1 WTDRDQBEARUVNC-LURJTMIESA-N 0.000 description 9
- WTDRDQBEARUVNC-UHFFFAOYSA-N L-Dopa Natural products OC(=O)C(N)CC1=CC=C(O)C(O)=C1 WTDRDQBEARUVNC-UHFFFAOYSA-N 0.000 description 9
- 208000025919 mucopolysaccharidosis type 7 Diseases 0.000 description 9
- 230000000626 neurodegenerative effect Effects 0.000 description 9
- 239000007787 solid Substances 0.000 description 9
- 206010003594 Ataxia telangiectasia Diseases 0.000 description 8
- 206010010904 Convulsion Diseases 0.000 description 8
- 241001465754 Metazoa Species 0.000 description 8
- 206010072928 Mucolipidosis type II Diseases 0.000 description 8
- 230000037396 body weight Effects 0.000 description 8
- 230000003111 delayed effect Effects 0.000 description 8
- HXWLAJVUJSVENX-HFIFKADTSA-N ioflupane I(123) Chemical compound C1([C@H]2C[C@@H]3CC[C@@H](N3CCCF)[C@H]2C(=O)OC)=CC=C([123I])C=C1 HXWLAJVUJSVENX-HFIFKADTSA-N 0.000 description 8
- HQKMJHAJHXVSDF-UHFFFAOYSA-L magnesium stearate Chemical compound [Mg+2].CCCCCCCCCCCCCCCCCC([O-])=O.CCCCCCCCCCCCCCCCCC([O-])=O HQKMJHAJHXVSDF-UHFFFAOYSA-L 0.000 description 8
- 230000009467 reduction Effects 0.000 description 8
- 206010003694 Atrophy Diseases 0.000 description 7
- 102000015554 Dopamine receptor Human genes 0.000 description 7
- 108050004812 Dopamine receptor Proteins 0.000 description 7
- 206010033799 Paralysis Diseases 0.000 description 7
- 208000007542 Paresis Diseases 0.000 description 7
- 230000037444 atrophy Effects 0.000 description 7
- 210000003169 central nervous system Anatomy 0.000 description 7
- 230000008859 change Effects 0.000 description 7
- 235000012000 cholesterol Nutrition 0.000 description 7
- 208000002173 dizziness Diseases 0.000 description 7
- 239000003937 drug carrier Substances 0.000 description 7
- 230000004424 eye movement Effects 0.000 description 7
- 210000003712 lysosome Anatomy 0.000 description 7
- 230000001868 lysosomic effect Effects 0.000 description 7
- 230000004060 metabolic process Effects 0.000 description 7
- 230000003565 oculomotor Effects 0.000 description 7
- 230000001575 pathological effect Effects 0.000 description 7
- 230000009747 swallowing Effects 0.000 description 7
- 208000019505 Deglutition disease Diseases 0.000 description 6
- 206010056696 Gaze palsy Diseases 0.000 description 6
- 206010021118 Hypotonia Diseases 0.000 description 6
- 208000007379 Muscle Hypotonia Diseases 0.000 description 6
- 208000010577 Niemann-Pick disease type C Diseases 0.000 description 6
- 229920002472 Starch Polymers 0.000 description 6
- 208000007930 Type C Niemann-Pick Disease Diseases 0.000 description 6
- -1 amine salts Chemical class 0.000 description 6
- 108010085377 beta-N-Acetylhexosaminidases Proteins 0.000 description 6
- 102000007478 beta-N-Acetylhexosaminidases Human genes 0.000 description 6
- 210000001638 cerebellum Anatomy 0.000 description 6
- 208000010877 cognitive disease Diseases 0.000 description 6
- 230000007850 degeneration Effects 0.000 description 6
- 208000035475 disorder Diseases 0.000 description 6
- 238000002651 drug therapy Methods 0.000 description 6
- 210000004744 fore-foot Anatomy 0.000 description 6
- 210000000548 hind-foot Anatomy 0.000 description 6
- 239000000546 pharmaceutical excipient Substances 0.000 description 6
- 239000000243 solution Substances 0.000 description 6
- 239000000725 suspension Substances 0.000 description 6
- 238000002560 therapeutic procedure Methods 0.000 description 6
- 208000016261 weight loss Diseases 0.000 description 6
- 206010068220 Aspartylglucosaminuria Diseases 0.000 description 5
- 206010067889 Dementia with Lewy bodies Diseases 0.000 description 5
- 208000024720 Fabry Disease Diseases 0.000 description 5
- 208000001308 Fasciculation Diseases 0.000 description 5
- 201000008892 GM1 Gangliosidosis Diseases 0.000 description 5
- 208000009796 Gangliosidoses Diseases 0.000 description 5
- 201000002832 Lewy body dementia Diseases 0.000 description 5
- 102000019305 Microtubule associated protein 1A Human genes 0.000 description 5
- 108050006673 Microtubule associated protein 1A Proteins 0.000 description 5
- 102000004866 Microtubule-associated protein 1B Human genes 0.000 description 5
- 108090001040 Microtubule-associated protein 1B Proteins 0.000 description 5
- 206010053694 Saccadic eye movement Diseases 0.000 description 5
- 201000007410 Smith-Lemli-Opitz syndrome Diseases 0.000 description 5
- 210000004027 cell Anatomy 0.000 description 5
- 230000001413 cellular effect Effects 0.000 description 5
- 230000007812 deficiency Effects 0.000 description 5
- 230000035622 drinking Effects 0.000 description 5
- 210000002683 foot Anatomy 0.000 description 5
- 229960004502 levodopa Drugs 0.000 description 5
- 230000015654 memory Effects 0.000 description 5
- 208000005264 motor neuron disease Diseases 0.000 description 5
- 230000035772 mutation Effects 0.000 description 5
- 230000001272 neurogenic effect Effects 0.000 description 5
- 208000021090 palsy Diseases 0.000 description 5
- 239000000843 powder Substances 0.000 description 5
- 230000004044 response Effects 0.000 description 5
- 208000002320 spinal muscular atrophy Diseases 0.000 description 5
- 210000001364 upper extremity Anatomy 0.000 description 5
- 230000004580 weight loss Effects 0.000 description 5
- 206010001541 Akinesia Diseases 0.000 description 4
- VTYYLEPIZMXCLO-UHFFFAOYSA-L Calcium carbonate Chemical compound [Ca+2].[O-]C([O-])=O VTYYLEPIZMXCLO-UHFFFAOYSA-L 0.000 description 4
- 208000022526 Canavan disease Diseases 0.000 description 4
- 208000002339 Frontotemporal Lobar Degeneration Diseases 0.000 description 4
- 208000001905 GM2 Gangliosidoses Diseases 0.000 description 4
- 208000015872 Gaucher disease Diseases 0.000 description 4
- 208000023105 Huntington disease Diseases 0.000 description 4
- 108010044467 Isoenzymes Proteins 0.000 description 4
- 201000011442 Metachromatic leukodystrophy Diseases 0.000 description 4
- 208000011644 Neurologic Gait disease Diseases 0.000 description 4
- 208000002537 Neuronal Ceroid-Lipofuscinoses Diseases 0.000 description 4
- 208000000609 Pick Disease of the Brain Diseases 0.000 description 4
- FTALBRSUTCGOEG-UHFFFAOYSA-N Riluzole Chemical compound C1=C(OC(F)(F)F)C=C2SC(N)=NC2=C1 FTALBRSUTCGOEG-UHFFFAOYSA-N 0.000 description 4
- 208000001163 Tangier disease Diseases 0.000 description 4
- 206010043189 Telangiectasia Diseases 0.000 description 4
- 208000018756 Variant Creutzfeldt-Jakob disease Diseases 0.000 description 4
- 230000002159 abnormal effect Effects 0.000 description 4
- 208000005881 bovine spongiform encephalopathy Diseases 0.000 description 4
- 210000004900 c-terminal fragment Anatomy 0.000 description 4
- 239000000969 carrier Substances 0.000 description 4
- 230000003920 cognitive function Effects 0.000 description 4
- 230000001934 delay Effects 0.000 description 4
- 201000006440 gangliosidosis Diseases 0.000 description 4
- 230000036541 health Effects 0.000 description 4
- 230000007774 longterm Effects 0.000 description 4
- 235000019359 magnesium stearate Nutrition 0.000 description 4
- 235000012054 meals Nutrition 0.000 description 4
- 230000004973 motor coordination Effects 0.000 description 4
- 201000006938 muscular dystrophy Diseases 0.000 description 4
- 201000003631 narcolepsy Diseases 0.000 description 4
- 230000001537 neural effect Effects 0.000 description 4
- 230000016273 neuron death Effects 0.000 description 4
- 238000007911 parenteral administration Methods 0.000 description 4
- 230000007170 pathology Effects 0.000 description 4
- 229960004181 riluzole Drugs 0.000 description 4
- 230000001148 spastic effect Effects 0.000 description 4
- 239000008107 starch Substances 0.000 description 4
- 235000019698 starch Nutrition 0.000 description 4
- 229940032147 starch Drugs 0.000 description 4
- 208000009056 telangiectasis Diseases 0.000 description 4
- WWUZIQQURGPMPG-UHFFFAOYSA-N (-)-D-erythro-Sphingosine Natural products CCCCCCCCCCCCCC=CC(O)C(N)CO WWUZIQQURGPMPG-UHFFFAOYSA-N 0.000 description 3
- 208000035657 Abasia Diseases 0.000 description 3
- 208000007848 Alcoholism Diseases 0.000 description 3
- 206010003062 Apraxia Diseases 0.000 description 3
- 206010003084 Areflexia Diseases 0.000 description 3
- OYPRJOBELJOOCE-UHFFFAOYSA-N Calcium Chemical compound [Ca] OYPRJOBELJOOCE-UHFFFAOYSA-N 0.000 description 3
- 206010012289 Dementia Diseases 0.000 description 3
- 208000012661 Dyskinesia Diseases 0.000 description 3
- 206010013952 Dysphonia Diseases 0.000 description 3
- 201000008905 GM2 gangliosidosis Diseases 0.000 description 3
- 108010010803 Gelatin Proteins 0.000 description 3
- 101150071246 Hexb gene Proteins 0.000 description 3
- 101001045440 Homo sapiens Beta-hexosaminidase subunit alpha Proteins 0.000 description 3
- 208000006083 Hypokinesia Diseases 0.000 description 3
- 208000016285 Movement disease Diseases 0.000 description 3
- 206010072927 Mucolipidosis type I Diseases 0.000 description 3
- 208000008238 Muscle Spasticity Diseases 0.000 description 3
- 101100459404 Neurospora crassa (strain ATCC 24698 / 74-OR23-1A / CBS 708.71 / DSM 1257 / FGSC 987) npc-1 gene Proteins 0.000 description 3
- 208000032319 Primary lateral sclerosis Diseases 0.000 description 3
- 208000024777 Prion disease Diseases 0.000 description 3
- 206010046298 Upper motor neurone lesion Diseases 0.000 description 3
- 230000001154 acute effect Effects 0.000 description 3
- 201000007930 alcohol dependence Diseases 0.000 description 3
- 230000015572 biosynthetic process Effects 0.000 description 3
- 229910052791 calcium Inorganic materials 0.000 description 3
- 239000011575 calcium Substances 0.000 description 3
- 230000001684 chronic effect Effects 0.000 description 3
- 230000019771 cognition Effects 0.000 description 3
- 150000001875 compounds Chemical class 0.000 description 3
- 238000007906 compression Methods 0.000 description 3
- 230000006835 compression Effects 0.000 description 3
- 208000006111 contracture Diseases 0.000 description 3
- 239000006071 cream Substances 0.000 description 3
- 210000003414 extremity Anatomy 0.000 description 3
- 210000001652 frontal lobe Anatomy 0.000 description 3
- GIVLTTJNORAZON-HDBOBKCLSA-N ganglioside GM2 (18:0) Chemical compound O[C@@H]1[C@@H](O)[C@H](OC[C@H](NC(=O)CCCCCCCCCCCCCCCCC)[C@H](O)\C=C\CCCCCCCCCCCCC)O[C@H](CO)[C@H]1O[C@H]1[C@H](O)[C@@H](O[C@]2(O[C@H]([C@H](NC(C)=O)[C@@H](O)C2)[C@H](O)[C@H](O)CO)C(O)=O)[C@@H](O[C@H]2[C@@H]([C@@H](O)[C@@H](O)[C@@H](CO)O2)NC(C)=O)[C@@H](CO)O1 GIVLTTJNORAZON-HDBOBKCLSA-N 0.000 description 3
- 239000008273 gelatin Substances 0.000 description 3
- 229920000159 gelatin Polymers 0.000 description 3
- 229940014259 gelatin Drugs 0.000 description 3
- 235000019322 gelatine Nutrition 0.000 description 3
- 235000011852 gelatine desserts Nutrition 0.000 description 3
- 210000003128 head Anatomy 0.000 description 3
- 206010020745 hyperreflexia Diseases 0.000 description 3
- 201000010901 lateral sclerosis Diseases 0.000 description 3
- 150000002632 lipids Chemical class 0.000 description 3
- 210000001259 mesencephalon Anatomy 0.000 description 3
- 230000000926 neurological effect Effects 0.000 description 3
- 210000002569 neuron Anatomy 0.000 description 3
- 230000003955 neuronal function Effects 0.000 description 3
- 201000001119 neuropathy Diseases 0.000 description 3
- 230000007823 neuropathy Effects 0.000 description 3
- 239000003921 oil Substances 0.000 description 3
- 235000019198 oils Nutrition 0.000 description 3
- 239000006186 oral dosage form Substances 0.000 description 3
- 201000008752 progressive muscular atrophy Diseases 0.000 description 3
- 208000026526 progressive weakness Diseases 0.000 description 3
- 108090000623 proteins and genes Proteins 0.000 description 3
- 230000007958 sleep Effects 0.000 description 3
- 208000026473 slurred speech Diseases 0.000 description 3
- 208000018198 spasticity Diseases 0.000 description 3
- WWUZIQQURGPMPG-KRWOKUGFSA-N sphingosine Chemical compound CCCCCCCCCCCCC\C=C\[C@@H](O)[C@@H](N)CO WWUZIQQURGPMPG-KRWOKUGFSA-N 0.000 description 3
- 230000008925 spontaneous activity Effects 0.000 description 3
- 208000023370 sporadic adult-onset ataxia of unknown etiology Diseases 0.000 description 3
- 239000008174 sterile solution Substances 0.000 description 3
- 230000004083 survival effect Effects 0.000 description 3
- 239000000375 suspending agent Substances 0.000 description 3
- 238000003786 synthesis reaction Methods 0.000 description 3
- 230000004462 vestibulo-ocular reflex Effects 0.000 description 3
- XLYOFNOQVPJJNP-UHFFFAOYSA-N water Substances O XLYOFNOQVPJJNP-UHFFFAOYSA-N 0.000 description 3
- NUKYPUAOHBNCPY-UHFFFAOYSA-N 4-aminopyridine Chemical compound NC1=CC=NC=C1 NUKYPUAOHBNCPY-UHFFFAOYSA-N 0.000 description 2
- 102100034215 AFG3-like protein 2 Human genes 0.000 description 2
- 208000011403 Alexander disease Diseases 0.000 description 2
- 208000031277 Amaurotic familial idiocy Diseases 0.000 description 2
- QGZKDVFQNNGYKY-UHFFFAOYSA-N Ammonia Chemical compound N QGZKDVFQNNGYKY-UHFFFAOYSA-N 0.000 description 2
- CIWBSHSKHKDKBQ-JLAZNSOCSA-N Ascorbic acid Chemical compound OC[C@H](O)[C@H]1OC(=O)C(O)=C1O CIWBSHSKHKDKBQ-JLAZNSOCSA-N 0.000 description 2
- 208000001827 Ataxia with vitamin E deficiency Diseases 0.000 description 2
- 102000007371 Ataxin-3 Human genes 0.000 description 2
- 208000031648 Body Weight Changes Diseases 0.000 description 2
- 206010006100 Bradykinesia Diseases 0.000 description 2
- 208000014644 Brain disease Diseases 0.000 description 2
- 206010068597 Bulbospinal muscular atrophy congenital Diseases 0.000 description 2
- 101100080277 Caenorhabditis elegans ncr-1 gene Proteins 0.000 description 2
- 208000010200 Cockayne syndrome Diseases 0.000 description 2
- 208000028698 Cognitive impairment Diseases 0.000 description 2
- 206010010305 Confusional state Diseases 0.000 description 2
- 208000032170 Congenital Abnormalities Diseases 0.000 description 2
- 208000020406 Creutzfeldt Jacob disease Diseases 0.000 description 2
- 208000003407 Creutzfeldt-Jakob Syndrome Diseases 0.000 description 2
- 208000010859 Creutzfeldt-Jakob disease Diseases 0.000 description 2
- 208000004986 Diffuse Cerebral Sclerosis of Schilder Diseases 0.000 description 2
- 206010049020 Encephalitis periaxialis diffusa Diseases 0.000 description 2
- 208000032274 Encephalopathy Diseases 0.000 description 2
- 208000019749 Eye movement disease Diseases 0.000 description 2
- 208000024412 Friedreich ataxia Diseases 0.000 description 2
- 208000010055 Globoid Cell Leukodystrophy Diseases 0.000 description 2
- 206010053185 Glycogen storage disease type II Diseases 0.000 description 2
- 101000780591 Homo sapiens AFG3-like protein 2 Proteins 0.000 description 2
- 206010020852 Hypertonia Diseases 0.000 description 2
- 206010021639 Incontinence Diseases 0.000 description 2
- 208000027747 Kennedy disease Diseases 0.000 description 2
- 208000028226 Krabbe disease Diseases 0.000 description 2
- GUBGYTABKSRVRQ-QKKXKWKRSA-N Lactose Natural products OC[C@H]1O[C@@H](O[C@H]2[C@H](O)[C@@H](O)C(O)O[C@@H]2CO)[C@H](O)[C@@H](O)[C@H]1O GUBGYTABKSRVRQ-QKKXKWKRSA-N 0.000 description 2
- 201000009035 MERRF syndrome Diseases 0.000 description 2
- 208000002569 Machado-Joseph Disease Diseases 0.000 description 2
- 208000026072 Motor neurone disease Diseases 0.000 description 2
- 208000002678 Mucopolysaccharidoses Diseases 0.000 description 2
- 206010028095 Mucopolysaccharidosis IV Diseases 0.000 description 2
- 208000000149 Multiple Sulfatase Deficiency Disease Diseases 0.000 description 2
- 208000035032 Multiple sulfatase deficiency Diseases 0.000 description 2
- 206010060860 Neurological symptom Diseases 0.000 description 2
- 208000015914 Non-Hodgkin lymphomas Diseases 0.000 description 2
- 208000036700 Oculomotor apraxia Diseases 0.000 description 2
- 208000019851 Opitz G/BBB syndrome Diseases 0.000 description 2
- 208000002193 Pain Diseases 0.000 description 2
- 208000031845 Pernicious anaemia Diseases 0.000 description 2
- 206010034701 Peroneal nerve palsy Diseases 0.000 description 2
- 208000009077 Pigmented Nevus Diseases 0.000 description 2
- GLUUGHFHXGJENI-UHFFFAOYSA-N Piperazine Chemical compound C1CNCCN1 GLUUGHFHXGJENI-UHFFFAOYSA-N 0.000 description 2
- 208000028017 Psychotic disease Diseases 0.000 description 2
- 208000005587 Refsum Disease Diseases 0.000 description 2
- 206010039424 Salivary hypersecretion Diseases 0.000 description 2
- 208000021235 Schilder disease Diseases 0.000 description 2
- 208000008630 Sialorrhea Diseases 0.000 description 2
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 description 2
- 208000036834 Spinocerebellar ataxia type 3 Diseases 0.000 description 2
- 206010041660 Splenomegaly Diseases 0.000 description 2
- 206010047571 Visual impairment Diseases 0.000 description 2
- 208000006269 X-Linked Bulbo-Spinal Atrophy Diseases 0.000 description 2
- 240000008042 Zea mays Species 0.000 description 2
- 235000005824 Zea mays ssp. parviglumis Nutrition 0.000 description 2
- 235000002017 Zea mays subsp mays Nutrition 0.000 description 2
- 239000002253 acid Substances 0.000 description 2
- 208000030597 adult Refsum disease Diseases 0.000 description 2
- VREFGVBLTWBCJP-UHFFFAOYSA-N alprazolam Chemical compound C12=CC(Cl)=CC=C2N2C(C)=NN=C2CN=C1C1=CC=CC=C1 VREFGVBLTWBCJP-UHFFFAOYSA-N 0.000 description 2
- 238000010171 animal model Methods 0.000 description 2
- 230000036528 appetite Effects 0.000 description 2
- 235000019789 appetite Nutrition 0.000 description 2
- 210000004957 autophagosome Anatomy 0.000 description 2
- 208000022778 autosomal recessive ataxia due to ubiquinone deficiency Diseases 0.000 description 2
- 230000003542 behavioural effect Effects 0.000 description 2
- 230000002146 bilateral effect Effects 0.000 description 2
- 239000011230 binding agent Substances 0.000 description 2
- 230000004579 body weight change Effects 0.000 description 2
- 210000000133 brain stem Anatomy 0.000 description 2
- 239000000872 buffer Substances 0.000 description 2
- 229910000019 calcium carbonate Inorganic materials 0.000 description 2
- 239000002775 capsule Substances 0.000 description 2
- 239000001913 cellulose Substances 0.000 description 2
- 235000010980 cellulose Nutrition 0.000 description 2
- 229920002678 cellulose Polymers 0.000 description 2
- 235000013339 cereals Nutrition 0.000 description 2
- 206010008129 cerebral palsy Diseases 0.000 description 2
- 239000003795 chemical substances by application Substances 0.000 description 2
- 210000000038 chest Anatomy 0.000 description 2
- CRQQGFGUEAVUIL-UHFFFAOYSA-N chlorothalonil Chemical compound ClC1=C(Cl)C(C#N)=C(Cl)C(C#N)=C1Cl CRQQGFGUEAVUIL-UHFFFAOYSA-N 0.000 description 2
- 210000000349 chromosome Anatomy 0.000 description 2
- 201000011228 clubfoot Diseases 0.000 description 2
- 230000007278 cognition impairment Effects 0.000 description 2
- 235000005822 corn Nutrition 0.000 description 2
- 230000002950 deficient Effects 0.000 description 2
- 239000003085 diluting agent Substances 0.000 description 2
- BNIILDVGGAEEIG-UHFFFAOYSA-L disodium hydrogen phosphate Chemical compound [Na+].[Na+].OP([O-])([O-])=O BNIILDVGGAEEIG-UHFFFAOYSA-L 0.000 description 2
- 229910000397 disodium phosphate Inorganic materials 0.000 description 2
- 235000019800 disodium phosphate Nutrition 0.000 description 2
- 239000002552 dosage form Substances 0.000 description 2
- 239000000975 dye Substances 0.000 description 2
- 239000003995 emulsifying agent Substances 0.000 description 2
- 239000000839 emulsion Substances 0.000 description 2
- 210000001163 endosome Anatomy 0.000 description 2
- 230000001747 exhibiting effect Effects 0.000 description 2
- 229960004979 fampridine Drugs 0.000 description 2
- 239000000796 flavoring agent Substances 0.000 description 2
- 235000013355 food flavoring agent Nutrition 0.000 description 2
- 235000003599 food sweetener Nutrition 0.000 description 2
- 238000009472 formulation Methods 0.000 description 2
- 150000002270 gangliosides Chemical class 0.000 description 2
- 239000000499 gel Substances 0.000 description 2
- 239000011521 glass Substances 0.000 description 2
- 230000004886 head movement Effects 0.000 description 2
- 230000000977 initiatory effect Effects 0.000 description 2
- 208000017476 juvenile neuronal ceroid lipofuscinosis Diseases 0.000 description 2
- 239000008101 lactose Substances 0.000 description 2
- 244000144972 livestock Species 0.000 description 2
- 239000011159 matrix material Substances 0.000 description 2
- 230000002438 mitochondrial effect Effects 0.000 description 2
- 201000007769 mucolipidosis Diseases 0.000 description 2
- 206010028093 mucopolysaccharidosis Diseases 0.000 description 2
- 208000022018 mucopolysaccharidosis type 2 Diseases 0.000 description 2
- 208000011045 mucopolysaccharidosis type 3 Diseases 0.000 description 2
- 210000003205 muscle Anatomy 0.000 description 2
- 201000008051 neuronal ceroid lipofuscinosis Diseases 0.000 description 2
- 201000007607 neuronal ceroid lipofuscinosis 3 Diseases 0.000 description 2
- 239000004090 neuroprotective agent Substances 0.000 description 2
- 208000028780 ocular motility disease Diseases 0.000 description 2
- 239000002674 ointment Substances 0.000 description 2
- 208000031237 olivopontocerebellar atrophy Diseases 0.000 description 2
- 230000002746 orthostatic effect Effects 0.000 description 2
- 230000001936 parietal effect Effects 0.000 description 2
- 230000001717 pathogenic effect Effects 0.000 description 2
- 208000033808 peripheral neuropathy Diseases 0.000 description 2
- 230000002085 persistent effect Effects 0.000 description 2
- 239000003755 preservative agent Substances 0.000 description 2
- 230000008569 process Effects 0.000 description 2
- 230000007101 progressive neurodegeneration Effects 0.000 description 2
- 102000004169 proteins and genes Human genes 0.000 description 2
- 230000017854 proteolysis Effects 0.000 description 2
- 230000011514 reflex Effects 0.000 description 2
- 230000000717 retained effect Effects 0.000 description 2
- 208000037118 sensory ataxia Diseases 0.000 description 2
- 230000003860 sleep quality Effects 0.000 description 2
- 239000011780 sodium chloride Substances 0.000 description 2
- 239000008247 solid mixture Substances 0.000 description 2
- 239000002904 solvent Substances 0.000 description 2
- 230000002269 spontaneous effect Effects 0.000 description 2
- 239000003381 stabilizer Substances 0.000 description 2
- 238000007619 statistical method Methods 0.000 description 2
- 238000013268 sustained release Methods 0.000 description 2
- 239000012730 sustained-release form Substances 0.000 description 2
- 239000003765 sweetening agent Substances 0.000 description 2
- 239000006188 syrup Substances 0.000 description 2
- 235000020357 syrup Nutrition 0.000 description 2
- 239000000454 talc Substances 0.000 description 2
- 229910052623 talc Inorganic materials 0.000 description 2
- 235000012222 talc Nutrition 0.000 description 2
- 230000001225 therapeutic effect Effects 0.000 description 2
- VZCYOOQTPOCHFL-UHFFFAOYSA-N trans-butenedioic acid Natural products OC(=O)C=CC(O)=O VZCYOOQTPOCHFL-UHFFFAOYSA-N 0.000 description 2
- 230000002792 vascular Effects 0.000 description 2
- 208000029257 vision disease Diseases 0.000 description 2
- 230000004304 visual acuity Effects 0.000 description 2
- 230000004393 visual impairment Effects 0.000 description 2
- 229940100445 wheat starch Drugs 0.000 description 2
- ZORQXIQZAOLNGE-UHFFFAOYSA-N 1,1-difluorocyclohexane Chemical compound FC1(F)CCCCC1 ZORQXIQZAOLNGE-UHFFFAOYSA-N 0.000 description 1
- NHBKXEKEPDILRR-UHFFFAOYSA-N 2,3-bis(butanoylsulfanyl)propyl butanoate Chemical compound CCCC(=O)OCC(SC(=O)CCC)CSC(=O)CCC NHBKXEKEPDILRR-UHFFFAOYSA-N 0.000 description 1
- HIQIXEFWDLTDED-UHFFFAOYSA-N 4-hydroxy-1-piperidin-4-ylpyrrolidin-2-one Chemical compound O=C1CC(O)CN1C1CCNCC1 HIQIXEFWDLTDED-UHFFFAOYSA-N 0.000 description 1
- HBAQYPYDRFILMT-UHFFFAOYSA-N 8-[3-(1-cyclopropylpyrazol-4-yl)-1H-pyrazolo[4,3-d]pyrimidin-5-yl]-3-methyl-3,8-diazabicyclo[3.2.1]octan-2-one Chemical class C1(CC1)N1N=CC(=C1)C1=NNC2=C1N=C(N=C2)N1C2C(N(CC1CC2)C)=O HBAQYPYDRFILMT-UHFFFAOYSA-N 0.000 description 1
- 208000000187 Abnormal Reflex Diseases 0.000 description 1
- QTBSBXVTEAMEQO-UHFFFAOYSA-M Acetate Chemical compound CC([O-])=O QTBSBXVTEAMEQO-UHFFFAOYSA-M 0.000 description 1
- 208000024893 Acute lymphoblastic leukemia Diseases 0.000 description 1
- 208000014697 Acute lymphocytic leukaemia Diseases 0.000 description 1
- 208000029602 Alpha-N-acetylgalactosaminidase deficiency Diseases 0.000 description 1
- 102000013455 Amyloid beta-Peptides Human genes 0.000 description 1
- 108010090849 Amyloid beta-Peptides Proteins 0.000 description 1
- 208000019901 Anxiety disease Diseases 0.000 description 1
- 206010002942 Apathy Diseases 0.000 description 1
- 101100256257 Arabidopsis thaliana SCAR4 gene Proteins 0.000 description 1
- 235000003911 Arachis Nutrition 0.000 description 1
- 244000105624 Arachis hypogaea Species 0.000 description 1
- 201000007848 Arts syndrome Diseases 0.000 description 1
- 206010003805 Autism Diseases 0.000 description 1
- 208000020706 Autistic disease Diseases 0.000 description 1
- 206010003840 Autonomic nervous system imbalance Diseases 0.000 description 1
- 208000012566 Autosomal recessive ataxia, Beauce type Diseases 0.000 description 1
- 208000033393 Autosomal recessive cerebellar ataxia-movement disorder syndrome Diseases 0.000 description 1
- 208000021768 Autosomal recessive spastic ataxia Diseases 0.000 description 1
- 208000035555 Beauce type autosomal recessive ataxia Diseases 0.000 description 1
- 208000001111 Bilateral Vestibulopathy Diseases 0.000 description 1
- 241000283690 Bos taurus Species 0.000 description 1
- FERIUCNNQQJTOY-UHFFFAOYSA-M Butyrate Chemical compound CCCC([O-])=O FERIUCNNQQJTOY-UHFFFAOYSA-M 0.000 description 1
- FERIUCNNQQJTOY-UHFFFAOYSA-N Butyric acid Natural products CCCC(O)=O FERIUCNNQQJTOY-UHFFFAOYSA-N 0.000 description 1
- KPIKSXCBFPERAY-FJXQXJEOSA-N C(C)(=O)NC(CC(C)C)C(=O)O.C(C)(=O)N[C@@H](CC(C)C)C(=O)O Chemical compound C(C)(=O)NC(CC(C)C)C(=O)O.C(C)(=O)N[C@@H](CC(C)C)C(=O)O KPIKSXCBFPERAY-FJXQXJEOSA-N 0.000 description 1
- YDNKGFDKKRUKPY-JHOUSYSJSA-N C16 ceramide Natural products CCCCCCCCCCCCCCCC(=O)N[C@@H](CO)[C@H](O)C=CCCCCCCCCCCCCC YDNKGFDKKRUKPY-JHOUSYSJSA-N 0.000 description 1
- 241000282472 Canis lupus familiaris Species 0.000 description 1
- 241000700198 Cavia Species 0.000 description 1
- 208000015879 Cerebellar disease Diseases 0.000 description 1
- 206010008072 Cerebellar syndrome Diseases 0.000 description 1
- 201000006867 Charcot-Marie-Tooth disease type 4 Diseases 0.000 description 1
- 208000035374 Chronic visceral acid sphingomyelinase deficiency Diseases 0.000 description 1
- KRKNYBCHXYNGOX-UHFFFAOYSA-K Citrate Chemical compound [O-]C(=O)CC(O)(CC([O-])=O)C([O-])=O KRKNYBCHXYNGOX-UHFFFAOYSA-K 0.000 description 1
- 208000033647 Classic progressive supranuclear palsy syndrome Diseases 0.000 description 1
- 208000010354 Coenzyme Q10 deficiency Diseases 0.000 description 1
- 208000027691 Conduct disease Diseases 0.000 description 1
- 206010056370 Congestive cardiomyopathy Diseases 0.000 description 1
- 208000016270 Corticobasal syndrome Diseases 0.000 description 1
- 208000014311 Cushing syndrome Diseases 0.000 description 1
- FBPFZTCFMRRESA-FSIIMWSLSA-N D-Glucitol Natural products OC[C@H](O)[C@H](O)[C@@H](O)[C@H](O)CO FBPFZTCFMRRESA-FSIIMWSLSA-N 0.000 description 1
- 208000011518 Danon disease Diseases 0.000 description 1
- 229920001353 Dextrin Polymers 0.000 description 1
- 239000004375 Dextrin Substances 0.000 description 1
- FEWJPZIEWOKRBE-JCYAYHJZSA-N Dextrotartaric acid Chemical compound OC(=O)[C@H](O)[C@@H](O)C(O)=O FEWJPZIEWOKRBE-JCYAYHJZSA-N 0.000 description 1
- 201000010046 Dilated cardiomyopathy Diseases 0.000 description 1
- 208000035762 Disorder of lipid metabolism Diseases 0.000 description 1
- 208000014094 Dystonic disease Diseases 0.000 description 1
- LVGKNOAMLMIIKO-UHFFFAOYSA-N Elaidinsaeure-aethylester Natural products CCCCCCCCC=CCCCCCCCC(=O)OCC LVGKNOAMLMIIKO-UHFFFAOYSA-N 0.000 description 1
- 102000004190 Enzymes Human genes 0.000 description 1
- 108090000790 Enzymes Proteins 0.000 description 1
- 241000283086 Equidae Species 0.000 description 1
- 208000010228 Erectile Dysfunction Diseases 0.000 description 1
- IAYPIBMASNFSPL-UHFFFAOYSA-N Ethylene oxide Chemical compound C1CO1 IAYPIBMASNFSPL-UHFFFAOYSA-N 0.000 description 1
- PIICEJLVQHRZGT-UHFFFAOYSA-N Ethylenediamine Chemical compound NCCN PIICEJLVQHRZGT-UHFFFAOYSA-N 0.000 description 1
- 206010015727 Extensor plantar response Diseases 0.000 description 1
- 241000282326 Felis catus Species 0.000 description 1
- 208000004067 Flatfoot Diseases 0.000 description 1
- VZCYOOQTPOCHFL-OWOJBTEDSA-N Fumaric acid Chemical compound OC(=O)\C=C\C(O)=O VZCYOOQTPOCHFL-OWOJBTEDSA-N 0.000 description 1
- 208000005622 Gait Ataxia Diseases 0.000 description 1
- 208000017462 Galactosialidosis Diseases 0.000 description 1
- 206010060891 General symptom Diseases 0.000 description 1
- 208000034826 Genetic Predisposition to Disease Diseases 0.000 description 1
- 208000003736 Gerstmann-Straussler-Scheinker Disease Diseases 0.000 description 1
- 206010072075 Gerstmann-Straussler-Scheinker syndrome Diseases 0.000 description 1
- WQZGKKKJIJFFOK-GASJEMHNSA-N Glucose Natural products OC[C@H]1OC(O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-GASJEMHNSA-N 0.000 description 1
- 208000001500 Glycogen Storage Disease Type IIb Diseases 0.000 description 1
- 208000035148 Glycogen storage disease due to LAMP-2 deficiency Diseases 0.000 description 1
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 description 1
- 244000060234 Gmelina philippensis Species 0.000 description 1
- 208000004547 Hallucinations Diseases 0.000 description 1
- 206010064950 Head titubation Diseases 0.000 description 1
- 208000006411 Hereditary Sensory and Motor Neuropathy Diseases 0.000 description 1
- 101150016456 Hexa gene Proteins 0.000 description 1
- 101000587058 Homo sapiens Methylenetetrahydrofolate reductase Proteins 0.000 description 1
- VEXZGXHMUGYJMC-UHFFFAOYSA-N Hydrochloric acid Chemical compound Cl VEXZGXHMUGYJMC-UHFFFAOYSA-N 0.000 description 1
- 208000035150 Hypercholesterolemia Diseases 0.000 description 1
- 206010021034 Hypometabolism Diseases 0.000 description 1
- 206010021089 Hyporeflexia Diseases 0.000 description 1
- DGAQECJNVWCQMB-PUAWFVPOSA-M Ilexoside XXIX Chemical compound C[C@@H]1CC[C@@]2(CC[C@@]3(C(=CC[C@H]4[C@]3(CC[C@@H]5[C@@]4(CC[C@@H](C5(C)C)OS(=O)(=O)[O-])C)C)[C@@H]2[C@]1(C)O)C)C(=O)O[C@H]6[C@@H]([C@H]([C@@H]([C@H](O6)CO)O)O)O.[Na+] DGAQECJNVWCQMB-PUAWFVPOSA-M 0.000 description 1
- 102000008394 Immunoglobulin Fragments Human genes 0.000 description 1
- 108010021625 Immunoglobulin Fragments Proteins 0.000 description 1
- 206010062016 Immunosuppression Diseases 0.000 description 1
- 206010021703 Indifference Diseases 0.000 description 1
- 208000035343 Infantile neurovisceral acid sphingomyelinase deficiency Diseases 0.000 description 1
- 206010023509 Kyphosis Diseases 0.000 description 1
- 241001313288 Labia Species 0.000 description 1
- JVTAAEKCZFNVCJ-UHFFFAOYSA-M Lactate Chemical compound CC(O)C([O-])=O JVTAAEKCZFNVCJ-UHFFFAOYSA-M 0.000 description 1
- 208000037795 Lethal ataxia with deafness and optic atrophy Diseases 0.000 description 1
- 206010024264 Lethargy Diseases 0.000 description 1
- 240000007472 Leucaena leucocephala Species 0.000 description 1
- 235000010643 Leucaena leucocephala Nutrition 0.000 description 1
- WHXSMMKQMYFTQS-UHFFFAOYSA-N Lithium Chemical compound [Li] WHXSMMKQMYFTQS-UHFFFAOYSA-N 0.000 description 1
- 208000016604 Lyme disease Diseases 0.000 description 1
- 206010025219 Lymphangioma Diseases 0.000 description 1
- 102100033448 Lysosomal alpha-glucosidase Human genes 0.000 description 1
- FYYHWMGAXLPEAU-UHFFFAOYSA-N Magnesium Chemical compound [Mg] FYYHWMGAXLPEAU-UHFFFAOYSA-N 0.000 description 1
- 241000124008 Mammalia Species 0.000 description 1
- 208000027382 Mental deterioration Diseases 0.000 description 1
- 206010027374 Mental impairment Diseases 0.000 description 1
- 208000036626 Mental retardation Diseases 0.000 description 1
- 102100029684 Methylenetetrahydrofolate reductase Human genes 0.000 description 1
- 229920000168 Microcrystalline cellulose Polymers 0.000 description 1
- 208000019695 Migraine disease Diseases 0.000 description 1
- 208000034819 Mobility Limitation Diseases 0.000 description 1
- 206010061296 Motor dysfunction Diseases 0.000 description 1
- 206010056893 Mucopolysaccharidosis VII Diseases 0.000 description 1
- 208000025797 Mucopolysaccharidosis type 4A Diseases 0.000 description 1
- 208000025923 Mucopolysaccharidosis type 4B Diseases 0.000 description 1
- 101100182721 Mus musculus Ly6e gene Proteins 0.000 description 1
- 208000010428 Muscle Weakness Diseases 0.000 description 1
- 208000021642 Muscular disease Diseases 0.000 description 1
- 206010028372 Muscular weakness Diseases 0.000 description 1
- 201000009623 Myopathy Diseases 0.000 description 1
- WXNXCEHXYPACJF-SSDOTTSWSA-N N-acetyl-D-leucine Chemical compound CC(C)C[C@H](C(O)=O)NC(C)=O WXNXCEHXYPACJF-SSDOTTSWSA-N 0.000 description 1
- CRJGESKKUOMBCT-VQTJNVASSA-N N-acetylsphinganine Chemical compound CCCCCCCCCCCCCCC[C@@H](O)[C@H](CO)NC(C)=O CRJGESKKUOMBCT-VQTJNVASSA-N 0.000 description 1
- MBBZMMPHUWSWHV-BDVNFPICSA-N N-methylglucamine Chemical compound CNC[C@H](O)[C@@H](O)[C@H](O)[C@H](O)CO MBBZMMPHUWSWHV-BDVNFPICSA-N 0.000 description 1
- 102100023306 Nesprin-1 Human genes 0.000 description 1
- 101710202335 Nesprin-1 Proteins 0.000 description 1
- 206010052057 Neuroborreliosis Diseases 0.000 description 1
- 208000027626 Neurocognitive disease Diseases 0.000 description 1
- 206010029333 Neurosis Diseases 0.000 description 1
- 201000000794 Niemann-Pick disease type A Diseases 0.000 description 1
- 201000000791 Niemann-Pick disease type B Diseases 0.000 description 1
- 208000000224 Night Terrors Diseases 0.000 description 1
- 206010031127 Orthostatic hypotension Diseases 0.000 description 1
- 241000283973 Oryctolagus cuniculus Species 0.000 description 1
- 206010052087 Oscillopsia Diseases 0.000 description 1
- 206010033892 Paraplegia Diseases 0.000 description 1
- 208000006199 Parasomnias Diseases 0.000 description 1
- 241001494479 Pecora Species 0.000 description 1
- 206010034719 Personality change Diseases 0.000 description 1
- ZLMJMSJWJFRBEC-UHFFFAOYSA-N Potassium Chemical compound [K] ZLMJMSJWJFRBEC-UHFFFAOYSA-N 0.000 description 1
- 208000006664 Precursor Cell Lymphoblastic Leukemia-Lymphoma Diseases 0.000 description 1
- 102000029797 Prion Human genes 0.000 description 1
- 108091000054 Prion Proteins 0.000 description 1
- 206010037180 Psychiatric symptoms Diseases 0.000 description 1
- 206010037213 Psychomotor retardation Diseases 0.000 description 1
- 208000034541 Rare lymphatic malformation Diseases 0.000 description 1
- 241000700159 Rattus Species 0.000 description 1
- 208000007014 Retinitis pigmentosa Diseases 0.000 description 1
- 101150093792 SPG7 gene Proteins 0.000 description 1
- 208000013608 Salla disease Diseases 0.000 description 1
- 208000025820 Sanfilippo syndrome type B Diseases 0.000 description 1
- 208000000828 Sialic Acid Storage Disease Diseases 0.000 description 1
- VYPSYNLAJGMNEJ-UHFFFAOYSA-N Silicium dioxide Chemical compound O=[Si]=O VYPSYNLAJGMNEJ-UHFFFAOYSA-N 0.000 description 1
- 206010041010 Sleep terror Diseases 0.000 description 1
- 241000282887 Suidae Species 0.000 description 1
- 102000005262 Sulfatase Human genes 0.000 description 1
- QAOWNCQODCNURD-UHFFFAOYSA-L Sulfate Chemical compound [O-]S([O-])(=O)=O QAOWNCQODCNURD-UHFFFAOYSA-L 0.000 description 1
- 206010048327 Supranuclear palsy Diseases 0.000 description 1
- 102000004243 Tubulin Human genes 0.000 description 1
- 108090000704 Tubulin Proteins 0.000 description 1
- 208000007824 Type A Niemann-Pick Disease Diseases 0.000 description 1
- 208000008291 Type B Niemann-Pick Disease Diseases 0.000 description 1
- 108700001567 Type I Schindler Disease Proteins 0.000 description 1
- 208000000921 Urge Urinary Incontinence Diseases 0.000 description 1
- 206010046543 Urinary incontinence Diseases 0.000 description 1
- 241000469816 Varus Species 0.000 description 1
- 206010068100 Vascular parkinsonism Diseases 0.000 description 1
- 241000251539 Vertebrata <Metazoa> Species 0.000 description 1
- 206010047642 Vitiligo Diseases 0.000 description 1
- 206010047700 Vomiting Diseases 0.000 description 1
- 206010067628 Winged scapula Diseases 0.000 description 1
- 208000006756 X-linked sideroblastic anemia Diseases 0.000 description 1
- 208000022440 X-linked sideroblastic anemia 1 Diseases 0.000 description 1
- 208000014938 X-linked spinocerebellar ataxia 1 Diseases 0.000 description 1
- 208000014790 X-linked spinocerebellar ataxia 2 Diseases 0.000 description 1
- 208000014787 X-linked spinocerebellar ataxia 3 Diseases 0.000 description 1
- 208000014788 X-linked spinocerebellar ataxia 4 Diseases 0.000 description 1
- 208000014789 X-linked spinocerebellar ataxia 5 Diseases 0.000 description 1
- HCHKCACWOHOZIP-UHFFFAOYSA-N Zinc Chemical compound [Zn] HCHKCACWOHOZIP-UHFFFAOYSA-N 0.000 description 1
- 230000005856 abnormality Effects 0.000 description 1
- DPXJVFZANSGRMM-UHFFFAOYSA-N acetic acid;2,3,4,5,6-pentahydroxyhexanal;sodium Chemical compound [Na].CC(O)=O.OCC(O)C(O)C(O)C(O)C=O DPXJVFZANSGRMM-UHFFFAOYSA-N 0.000 description 1
- 230000002378 acidificating effect Effects 0.000 description 1
- 150000007513 acids Chemical class 0.000 description 1
- 230000009471 action Effects 0.000 description 1
- 230000004913 activation Effects 0.000 description 1
- 239000000654 additive Substances 0.000 description 1
- 230000002411 adverse Effects 0.000 description 1
- 239000000443 aerosol Substances 0.000 description 1
- 238000013019 agitation Methods 0.000 description 1
- 150000001298 alcohols Chemical class 0.000 description 1
- 229910052783 alkali metal Inorganic materials 0.000 description 1
- 229910052784 alkaline earth metal Inorganic materials 0.000 description 1
- 150000003973 alkyl amines Chemical class 0.000 description 1
- SHZGCJCMOBCMKK-PHYPRBDBSA-N alpha-D-fucose Chemical compound C[C@H]1O[C@H](O)[C@H](O)[C@@H](O)[C@H]1O SHZGCJCMOBCMKK-PHYPRBDBSA-N 0.000 description 1
- 102000005840 alpha-Galactosidase Human genes 0.000 description 1
- 108010030291 alpha-Galactosidase Proteins 0.000 description 1
- 229910052782 aluminium Inorganic materials 0.000 description 1
- XAGFODPZIPBFFR-UHFFFAOYSA-N aluminium Chemical compound [Al] XAGFODPZIPBFFR-UHFFFAOYSA-N 0.000 description 1
- 230000007792 alzheimer disease pathology Effects 0.000 description 1
- 229960000212 aminophenazone Drugs 0.000 description 1
- 229910021529 ammonia Inorganic materials 0.000 description 1
- 229960000510 ammonia Drugs 0.000 description 1
- 150000008064 anhydrides Chemical class 0.000 description 1
- 239000003920 antivertigo agent Substances 0.000 description 1
- 230000036506 anxiety Effects 0.000 description 1
- 239000007864 aqueous solution Substances 0.000 description 1
- 229940072107 ascorbate Drugs 0.000 description 1
- 235000010323 ascorbic acid Nutrition 0.000 description 1
- 239000011668 ascorbic acid Substances 0.000 description 1
- 230000003935 attention Effects 0.000 description 1
- 230000004900 autophagic degradation Effects 0.000 description 1
- 230000002886 autophagic effect Effects 0.000 description 1
- 230000004908 autophagic flux Effects 0.000 description 1
- 230000005033 autophagosome formation Effects 0.000 description 1
- 208000027562 autosomal recessive cerebellar ataxia-saccadic intrusion syndrome Diseases 0.000 description 1
- 208000021024 autosomal recessive inheritance Diseases 0.000 description 1
- 208000015988 autosomal recessive spinocerebellar ataxia 4 Diseases 0.000 description 1
- 208000015991 autosomal recessive spinocerebellar ataxia 8 Diseases 0.000 description 1
- 229910052788 barium Inorganic materials 0.000 description 1
- DSAJWYNOEDNPEQ-UHFFFAOYSA-N barium atom Chemical compound [Ba] DSAJWYNOEDNPEQ-UHFFFAOYSA-N 0.000 description 1
- 238000010009 beating Methods 0.000 description 1
- 238000005452 bending Methods 0.000 description 1
- 230000009286 beneficial effect Effects 0.000 description 1
- UPABQMWFWCMOFV-UHFFFAOYSA-N benethamine Chemical compound C=1C=CC=CC=1CNCCC1=CC=CC=C1 UPABQMWFWCMOFV-UHFFFAOYSA-N 0.000 description 1
- JUHORIMYRDESRB-UHFFFAOYSA-N benzathine Chemical compound C=1C=CC=CC=1CNCCNCC1=CC=CC=C1 JUHORIMYRDESRB-UHFFFAOYSA-N 0.000 description 1
- WQZGKKKJIJFFOK-VFUOTHLCSA-N beta-D-glucose Chemical compound OC[C@H]1O[C@@H](O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-VFUOTHLCSA-N 0.000 description 1
- 201000006486 beta-mannosidosis Diseases 0.000 description 1
- 239000003833 bile salt Substances 0.000 description 1
- 229940093761 bile salts Drugs 0.000 description 1
- 206010005159 blepharospasm Diseases 0.000 description 1
- 230000000744 blepharospasm Effects 0.000 description 1
- 235000021152 breakfast Nutrition 0.000 description 1
- 239000006172 buffering agent Substances 0.000 description 1
- 235000010216 calcium carbonate Nutrition 0.000 description 1
- FUFJGUQYACFECW-UHFFFAOYSA-L calcium hydrogenphosphate Chemical compound [Ca+2].OP([O-])([O-])=O FUFJGUQYACFECW-UHFFFAOYSA-L 0.000 description 1
- 239000001506 calcium phosphate Substances 0.000 description 1
- 229910000389 calcium phosphate Inorganic materials 0.000 description 1
- 235000011010 calcium phosphates Nutrition 0.000 description 1
- 230000003185 calcium uptake Effects 0.000 description 1
- 238000004364 calculation method Methods 0.000 description 1
- 239000001768 carboxy methyl cellulose Substances 0.000 description 1
- 230000015556 catabolic process Effects 0.000 description 1
- 230000001364 causal effect Effects 0.000 description 1
- 239000002771 cell marker Substances 0.000 description 1
- 230000004640 cellular pathway Effects 0.000 description 1
- 229940106189 ceramide Drugs 0.000 description 1
- ZVEQCJWYRWKARO-UHFFFAOYSA-N ceramide Natural products CCCCCCCCCCCCCCC(O)C(=O)NC(CO)C(O)C=CCCC=C(C)CCCCCCCCC ZVEQCJWYRWKARO-UHFFFAOYSA-N 0.000 description 1
- 208000034405 cerebellar type multiple system atrophy Diseases 0.000 description 1
- VDANGULDQQJODZ-UHFFFAOYSA-N chloroprocaine Chemical compound CCN(CC)CCOC(=O)C1=CC=C(N)C=C1Cl VDANGULDQQJODZ-UHFFFAOYSA-N 0.000 description 1
- 229960002023 chloroprocaine Drugs 0.000 description 1
- 201000001883 cholelithiasis Diseases 0.000 description 1
- 102000049842 cholesterol binding protein Human genes 0.000 description 1
- 108010011793 cholesterol binding protein Proteins 0.000 description 1
- OEYIOHPDSNJKLS-UHFFFAOYSA-N choline Chemical compound C[N+](C)(C)CCO OEYIOHPDSNJKLS-UHFFFAOYSA-N 0.000 description 1
- 229960001231 choline Drugs 0.000 description 1
- 208000017580 chronic wasting disease Diseases 0.000 description 1
- 229940001468 citrate Drugs 0.000 description 1
- 239000011248 coating agent Substances 0.000 description 1
- 238000000576 coating method Methods 0.000 description 1
- 235000019864 coconut oil Nutrition 0.000 description 1
- 239000003240 coconut oil Substances 0.000 description 1
- 229940075614 colloidal silicon dioxide Drugs 0.000 description 1
- 239000003086 colorant Substances 0.000 description 1
- 230000036461 convulsion Effects 0.000 description 1
- 210000004087 cornea Anatomy 0.000 description 1
- 201000007717 corneal ulcer Diseases 0.000 description 1
- 239000003246 corticosteroid Substances 0.000 description 1
- 238000009109 curative therapy Methods 0.000 description 1
- 230000009849 deactivation Effects 0.000 description 1
- 230000004452 decreased vision Effects 0.000 description 1
- 230000003412 degenerative effect Effects 0.000 description 1
- 230000001419 dependent effect Effects 0.000 description 1
- 238000001514 detection method Methods 0.000 description 1
- 235000019425 dextrin Nutrition 0.000 description 1
- 235000019700 dicalcium phosphate Nutrition 0.000 description 1
- ZBCBWPMODOFKDW-UHFFFAOYSA-N diethanolamine Chemical compound OCCNCCO ZBCBWPMODOFKDW-UHFFFAOYSA-N 0.000 description 1
- 229940043237 diethanolamine Drugs 0.000 description 1
- HPNMFZURTQLUMO-UHFFFAOYSA-N diethylamine Chemical compound CCNCC HPNMFZURTQLUMO-UHFFFAOYSA-N 0.000 description 1
- 230000008034 disappearance Effects 0.000 description 1
- 230000009266 disease activity Effects 0.000 description 1
- 208000010118 dystonia Diseases 0.000 description 1
- 230000000632 dystonic effect Effects 0.000 description 1
- 210000005069 ears Anatomy 0.000 description 1
- 230000002121 endocytic effect Effects 0.000 description 1
- 230000012202 endocytosis Effects 0.000 description 1
- 150000002148 esters Chemical class 0.000 description 1
- LVGKNOAMLMIIKO-QXMHVHEDSA-N ethyl oleate Chemical compound CCCCCCCC\C=C/CCCCCCCC(=O)OCC LVGKNOAMLMIIKO-QXMHVHEDSA-N 0.000 description 1
- 229940093471 ethyl oleate Drugs 0.000 description 1
- 238000011156 evaluation Methods 0.000 description 1
- 238000002474 experimental method Methods 0.000 description 1
- 238000000605 extraction Methods 0.000 description 1
- 230000004438 eyesight Effects 0.000 description 1
- 230000008921 facial expression Effects 0.000 description 1
- 201000006061 fatal familial insomnia Diseases 0.000 description 1
- 206010016256 fatigue Diseases 0.000 description 1
- 235000019197 fats Nutrition 0.000 description 1
- 208000037957 feline spongiform encephalopathy Diseases 0.000 description 1
- 230000001605 fetal effect Effects 0.000 description 1
- 239000000945 filler Substances 0.000 description 1
- 239000007850 fluorescent dye Substances 0.000 description 1
- 235000013305 food Nutrition 0.000 description 1
- 210000003194 forelimb Anatomy 0.000 description 1
- 239000012634 fragment Substances 0.000 description 1
- 230000004927 fusion Effects 0.000 description 1
- 230000003371 gabaergic effect Effects 0.000 description 1
- QPJBWNIQKHGLAU-IQZHVAEDSA-N ganglioside GM1 Chemical compound O[C@@H]1[C@@H](O)[C@H](OC[C@H](NC(=O)CCCCCCCCCCCCCCCCC)[C@H](O)\C=C\CCCCCCCCCCCCC)O[C@H](CO)[C@H]1O[C@H]1[C@H](O)[C@@H](O[C@]2(O[C@H]([C@H](NC(C)=O)[C@@H](O)C2)[C@H](O)[C@H](O)CO)C(O)=O)[C@@H](O[C@H]2[C@@H]([C@@H](O[C@H]3[C@@H]([C@@H](O)[C@@H](O)[C@@H](CO)O3)O)[C@@H](O)[C@@H](CO)O2)NC(C)=O)[C@@H](CO)O1 QPJBWNIQKHGLAU-IQZHVAEDSA-N 0.000 description 1
- 239000008103 glucose Substances 0.000 description 1
- 201000004502 glycogen storage disease II Diseases 0.000 description 1
- 150000002334 glycols Chemical class 0.000 description 1
- 239000008187 granular material Substances 0.000 description 1
- 208000035474 group of disease Diseases 0.000 description 1
- 150000008282 halocarbons Chemical class 0.000 description 1
- LNEPOXFFQSENCJ-UHFFFAOYSA-N haloperidol Chemical compound C1CC(O)(C=2C=CC(Cl)=CC=2)CCN1CCCC(=O)C1=CC=C(F)C=C1 LNEPOXFFQSENCJ-UHFFFAOYSA-N 0.000 description 1
- 208000007475 hemolytic anemia Diseases 0.000 description 1
- 208000021995 hereditary motor and sensory neuropathy Diseases 0.000 description 1
- 208000008675 hereditary spastic paraplegia Diseases 0.000 description 1
- 201000007110 hereditary spastic paraplegia 7 Diseases 0.000 description 1
- 238000004128 high performance liquid chromatography Methods 0.000 description 1
- 239000000017 hydrogel Substances 0.000 description 1
- 208000006278 hypochromic anemia Diseases 0.000 description 1
- 208000009300 hypochromic microcytic anemia Diseases 0.000 description 1
- 230000003483 hypokinetic effect Effects 0.000 description 1
- 230000002806 hypometabolic effect Effects 0.000 description 1
- 230000001506 immunosuppresive effect Effects 0.000 description 1
- 201000001881 impotence Diseases 0.000 description 1
- 238000001727 in vivo Methods 0.000 description 1
- 208000036545 infantile onset glycogen storage disease due to acid maltase deficiency Diseases 0.000 description 1
- 208000010194 infantile onset spinocerebellar ataxia Diseases 0.000 description 1
- 230000002757 inflammatory effect Effects 0.000 description 1
- 230000002401 inhibitory effect Effects 0.000 description 1
- 230000005764 inhibitory process Effects 0.000 description 1
- 229910052500 inorganic mineral Inorganic materials 0.000 description 1
- 230000003815 intracellular cholesterol transport Effects 0.000 description 1
- 238000007918 intramuscular administration Methods 0.000 description 1
- 238000007912 intraperitoneal administration Methods 0.000 description 1
- 238000007913 intrathecal administration Methods 0.000 description 1
- 238000010253 intravenous injection Methods 0.000 description 1
- 239000003456 ion exchange resin Substances 0.000 description 1
- 229920003303 ion-exchange polymer Polymers 0.000 description 1
- 206010023497 kuru Diseases 0.000 description 1
- 229960001375 lactose Drugs 0.000 description 1
- 230000002045 lasting effect Effects 0.000 description 1
- 208000035157 late-onset glycogen storage disease due to acid maltase deficiency Diseases 0.000 description 1
- 231100000518 lethal Toxicity 0.000 description 1
- 230000001665 lethal effect Effects 0.000 description 1
- 229940080381 levodopa 100 mg Drugs 0.000 description 1
- 208000027905 limb weakness Diseases 0.000 description 1
- 231100000861 limb weakness Toxicity 0.000 description 1
- 230000000670 limiting effect Effects 0.000 description 1
- 230000006372 lipid accumulation Effects 0.000 description 1
- 229910052744 lithium Inorganic materials 0.000 description 1
- 238000011068 loading method Methods 0.000 description 1
- 238000011866 long-term treatment Methods 0.000 description 1
- 208000018883 loss of balance Diseases 0.000 description 1
- 239000000314 lubricant Substances 0.000 description 1
- 229920002521 macromolecule Polymers 0.000 description 1
- 229910052749 magnesium Inorganic materials 0.000 description 1
- 239000011777 magnesium Substances 0.000 description 1
- 238000012423 maintenance Methods 0.000 description 1
- VZCYOOQTPOCHFL-UPHRSURJSA-N maleic acid Chemical compound OC(=O)\C=C/C(O)=O VZCYOOQTPOCHFL-UPHRSURJSA-N 0.000 description 1
- 239000000463 material Substances 0.000 description 1
- 238000002844 melting Methods 0.000 description 1
- 230000008018 melting Effects 0.000 description 1
- 239000012528 membrane Substances 0.000 description 1
- 230000003340 mental effect Effects 0.000 description 1
- 230000002503 metabolic effect Effects 0.000 description 1
- 239000002207 metabolite Substances 0.000 description 1
- 229910052751 metal Inorganic materials 0.000 description 1
- 239000002184 metal Substances 0.000 description 1
- 229940016286 microcrystalline cellulose Drugs 0.000 description 1
- 235000019813 microcrystalline cellulose Nutrition 0.000 description 1
- 239000008108 microcrystalline cellulose Substances 0.000 description 1
- 206010027599 migraine Diseases 0.000 description 1
- 239000011707 mineral Substances 0.000 description 1
- 201000011561 mitochondrial DNA depletion syndrome 7 Diseases 0.000 description 1
- 238000002156 mixing Methods 0.000 description 1
- 210000002864 mononuclear phagocyte Anatomy 0.000 description 1
- 230000008450 motivation Effects 0.000 description 1
- 210000000337 motor cortex Anatomy 0.000 description 1
- 230000008111 motor development Effects 0.000 description 1
- 201000005545 motor peripheral neuropathy Diseases 0.000 description 1
- 208000020460 mucolipidosis II alpha/beta Diseases 0.000 description 1
- 208000012227 mucopolysaccharidosis type IIIB Diseases 0.000 description 1
- 201000000585 muscular atrophy Diseases 0.000 description 1
- 210000001577 neostriatum Anatomy 0.000 description 1
- 230000009251 neurologic dysfunction Effects 0.000 description 1
- 208000015015 neurological dysfunction Diseases 0.000 description 1
- 238000010984 neurological examination Methods 0.000 description 1
- 230000006764 neuronal dysfunction Effects 0.000 description 1
- 230000003961 neuronal insult Effects 0.000 description 1
- 230000000324 neuroprotective effect Effects 0.000 description 1
- 230000003557 neuropsychological effect Effects 0.000 description 1
- 238000010855 neuropsychological testing Methods 0.000 description 1
- 208000015238 neurotic disease Diseases 0.000 description 1
- VVGIYYKRAMHVLU-UHFFFAOYSA-N newbouldiamide Natural products CCCCCCCCCCCCCCCCCCCC(O)C(O)C(O)C(CO)NC(=O)CCCCCCCCCCCCCCCCC VVGIYYKRAMHVLU-UHFFFAOYSA-N 0.000 description 1
- 231100000957 no side effect Toxicity 0.000 description 1
- 230000000422 nocturnal effect Effects 0.000 description 1
- 201000003077 normal pressure hydrocephalus Diseases 0.000 description 1
- 101150049361 npc-1 gene Proteins 0.000 description 1
- 101150107867 npc-2 gene Proteins 0.000 description 1
- 210000000869 occipital lobe Anatomy 0.000 description 1
- 229940049964 oleate Drugs 0.000 description 1
- 206010030875 ophthalmoplegia Diseases 0.000 description 1
- 210000003463 organelle Anatomy 0.000 description 1
- 150000007524 organic acids Chemical class 0.000 description 1
- 235000005985 organic acids Nutrition 0.000 description 1
- 239000003960 organic solvent Substances 0.000 description 1
- 230000000399 orthopedic effect Effects 0.000 description 1
- 231100000898 oscillopsia Toxicity 0.000 description 1
- 210000001152 parietal lobe Anatomy 0.000 description 1
- 230000036961 partial effect Effects 0.000 description 1
- 230000003950 pathogenic mechanism Effects 0.000 description 1
- 230000037361 pathway Effects 0.000 description 1
- 230000008447 perception Effects 0.000 description 1
- 230000002093 peripheral effect Effects 0.000 description 1
- 210000001428 peripheral nervous system Anatomy 0.000 description 1
- 230000000144 pharmacologic effect Effects 0.000 description 1
- 230000001830 phrenic effect Effects 0.000 description 1
- 239000006187 pill Substances 0.000 description 1
- 229920003023 plastic Polymers 0.000 description 1
- 239000004033 plastic Substances 0.000 description 1
- 239000000244 polyoxyethylene sorbitan monooleate Substances 0.000 description 1
- 235000010482 polyoxyethylene sorbitan monooleate Nutrition 0.000 description 1
- 229940068968 polysorbate 80 Drugs 0.000 description 1
- 229920000053 polysorbate 80 Polymers 0.000 description 1
- 229920006316 polyvinylpyrrolidine Polymers 0.000 description 1
- 229910052700 potassium Inorganic materials 0.000 description 1
- 239000011591 potassium Substances 0.000 description 1
- 239000002243 precursor Substances 0.000 description 1
- 238000002360 preparation method Methods 0.000 description 1
- 230000002335 preservative effect Effects 0.000 description 1
- 230000003518 presynaptic effect Effects 0.000 description 1
- 230000002265 prevention Effects 0.000 description 1
- 201000009395 primary hyperaldosteronism Diseases 0.000 description 1
- 239000013615 primer Substances 0.000 description 1
- 239000002987 primer (paints) Substances 0.000 description 1
- MFDFERRIHVXMIY-UHFFFAOYSA-N procaine Chemical compound CCN(CC)CCOC(=O)C1=CC=C(N)C=C1 MFDFERRIHVXMIY-UHFFFAOYSA-N 0.000 description 1
- 229960004919 procaine Drugs 0.000 description 1
- 208000032207 progressive 1 supranuclear palsy Diseases 0.000 description 1
- 239000003380 propellant Substances 0.000 description 1
- 230000000069 prophylactic effect Effects 0.000 description 1
- 230000000272 proprioceptive effect Effects 0.000 description 1
- 230000008132 psychomotor development Effects 0.000 description 1
- 210000000449 purkinje cell Anatomy 0.000 description 1
- 238000011002 quantification Methods 0.000 description 1
- 229940107591 quetiapine 25 mg Drugs 0.000 description 1
- 230000036385 rapid eye movement (rem) sleep Effects 0.000 description 1
- 238000011552 rat model Methods 0.000 description 1
- 230000000306 recurrent effect Effects 0.000 description 1
- 238000011160 research Methods 0.000 description 1
- UHSKFQJFRQCDBE-UHFFFAOYSA-N ropinirole Chemical compound CCCN(CCC)CCC1=CC=CC2=C1CC(=O)N2 UHSKFQJFRQCDBE-UHFFFAOYSA-N 0.000 description 1
- 229960001879 ropinirole Drugs 0.000 description 1
- 239000000523 sample Substances 0.000 description 1
- 231100000241 scar Toxicity 0.000 description 1
- 206010039722 scoliosis Diseases 0.000 description 1
- 208000008864 scrapie Diseases 0.000 description 1
- 230000001953 sensory effect Effects 0.000 description 1
- 201000007245 sideroblastic anemia 1 Diseases 0.000 description 1
- 238000009097 single-agent therapy Methods 0.000 description 1
- 208000019116 sleep disease Diseases 0.000 description 1
- 208000022925 sleep disturbance Diseases 0.000 description 1
- 230000004469 smooth pursuit movement Effects 0.000 description 1
- 230000003997 social interaction Effects 0.000 description 1
- 229910052708 sodium Inorganic materials 0.000 description 1
- 239000011734 sodium Substances 0.000 description 1
- 235000019812 sodium carboxymethyl cellulose Nutrition 0.000 description 1
- 229920001027 sodium carboxymethylcellulose Polymers 0.000 description 1
- 239000001488 sodium phosphate Substances 0.000 description 1
- 229920003109 sodium starch glycolate Polymers 0.000 description 1
- 229940079832 sodium starch glycolate Drugs 0.000 description 1
- 239000008109 sodium starch glycolate Substances 0.000 description 1
- 239000007909 solid dosage form Substances 0.000 description 1
- 239000001593 sorbitan monooleate Substances 0.000 description 1
- 235000011069 sorbitan monooleate Nutrition 0.000 description 1
- 229940035049 sorbitan monooleate Drugs 0.000 description 1
- 239000000600 sorbitol Substances 0.000 description 1
- 230000009870 specific binding Effects 0.000 description 1
- 150000003408 sphingolipids Chemical class 0.000 description 1
- 210000000278 spinal cord Anatomy 0.000 description 1
- 201000003570 spinocerebellar ataxia type 17 Diseases 0.000 description 1
- 239000007921 spray Substances 0.000 description 1
- 230000007411 stabilization of membrane potential Effects 0.000 description 1
- 239000008223 sterile water Substances 0.000 description 1
- 239000010902 straw Substances 0.000 description 1
- 208000023516 stroke disease Diseases 0.000 description 1
- 238000010254 subcutaneous injection Methods 0.000 description 1
- 239000007929 subcutaneous injection Substances 0.000 description 1
- 229940086735 succinate Drugs 0.000 description 1
- KDYFGRWQOYBRFD-UHFFFAOYSA-L succinate(2-) Chemical compound [O-]C(=O)CCC([O-])=O KDYFGRWQOYBRFD-UHFFFAOYSA-L 0.000 description 1
- 235000000346 sugar Nutrition 0.000 description 1
- 150000005846 sugar alcohols Polymers 0.000 description 1
- 108060007951 sulfatase Proteins 0.000 description 1
- 230000001629 suppression Effects 0.000 description 1
- 230000003976 synaptic dysfunction Effects 0.000 description 1
- 229940095064 tartrate Drugs 0.000 description 1
- 210000003478 temporal lobe Anatomy 0.000 description 1
- 230000000542 thalamic effect Effects 0.000 description 1
- 239000002562 thickening agent Substances 0.000 description 1
- 210000000115 thoracic cavity Anatomy 0.000 description 1
- 210000001519 tissue Anatomy 0.000 description 1
- 239000008181 tonicity modifier Substances 0.000 description 1
- 230000000699 topical effect Effects 0.000 description 1
- 231100000331 toxic Toxicity 0.000 description 1
- 230000002588 toxic effect Effects 0.000 description 1
- 230000001052 transient effect Effects 0.000 description 1
- 229910052723 transition metal Inorganic materials 0.000 description 1
- 102000035160 transmembrane proteins Human genes 0.000 description 1
- 108091005703 transmembrane proteins Proteins 0.000 description 1
- 208000037956 transmissible mink encephalopathy Diseases 0.000 description 1
- 230000032258 transport Effects 0.000 description 1
- QORWJWZARLRLPR-UHFFFAOYSA-H tricalcium bis(phosphate) Chemical compound [Ca+2].[Ca+2].[Ca+2].[O-]P([O-])([O-])=O.[O-]P([O-])([O-])=O QORWJWZARLRLPR-UHFFFAOYSA-H 0.000 description 1
- LENZDBCJOHFCAS-UHFFFAOYSA-N tris Chemical compound OCC(N)(CO)CO LENZDBCJOHFCAS-UHFFFAOYSA-N 0.000 description 1
- 206010046494 urge incontinence Diseases 0.000 description 1
- 210000003934 vacuole Anatomy 0.000 description 1
- 229940070710 valerate Drugs 0.000 description 1
- NQPDZGIKBAWPEJ-UHFFFAOYSA-N valeric acid Chemical compound CCCCC(O)=O NQPDZGIKBAWPEJ-UHFFFAOYSA-N 0.000 description 1
- 239000003981 vehicle Substances 0.000 description 1
- 201000000200 vestibular neuronitis Diseases 0.000 description 1
- 239000004034 viscosity adjusting agent Substances 0.000 description 1
- 230000008673 vomiting Effects 0.000 description 1
- 239000001993 wax Substances 0.000 description 1
- 230000004584 weight gain Effects 0.000 description 1
- 235000019786 weight gain Nutrition 0.000 description 1
- 230000036642 wellbeing Effects 0.000 description 1
- 239000000080 wetting agent Substances 0.000 description 1
- 208000032620 x-linked multiple congenital anomalies-neurodevelopmental syndrome Diseases 0.000 description 1
- 229910052725 zinc Inorganic materials 0.000 description 1
- 239000011701 zinc Substances 0.000 description 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/13—Amines
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/185—Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
- A61K31/19—Carboxylic acids, e.g. valproic acid
- A61K31/195—Carboxylic acids, e.g. valproic acid having an amino group
- A61K31/197—Carboxylic acids, e.g. valproic acid having an amino group the amino and the carboxyl groups being attached to the same acyclic carbon chain, e.g. gamma-aminobutyric acid [GABA], beta-alanine, epsilon-aminocaproic acid or pantothenic acid
- A61K31/198—Alpha-amino acids, e.g. alanine or edetic acid [EDTA]
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/04—Centrally acting analgesics, e.g. opioids
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/20—Hypnotics; Sedatives
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/30—Drugs for disorders of the nervous system for treating abuse or dependence
- A61P25/32—Alcohol-abuse
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
- A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
- C07C233/00—Carboxylic acid amides
- C07C233/01—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms
- C07C233/45—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by carboxyl groups
- C07C233/46—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by carboxyl groups with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Life Sciences & Earth Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Neurology (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Neurosurgery (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Epidemiology (AREA)
- Psychiatry (AREA)
- Psychology (AREA)
- Hospice & Palliative Care (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Immunology (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Ophthalmology & Optometry (AREA)
- Pain & Pain Management (AREA)
- Obesity (AREA)
- Addiction (AREA)
- Transplantation (AREA)
- Anesthesiology (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| KR1020257008819A KR20250042197A (ko) | 2016-08-11 | 2017-08-11 | 신경퇴행성 질병을 위한 치료제 |
Applications Claiming Priority (10)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GB1613829.9 | 2016-08-11 | ||
| GBGB1613829.9A GB201613829D0 (en) | 2016-08-11 | 2016-08-11 | Therapeutic agents for neurodegenerative diseases, lysosomal storage disorders and neurodegenerative diseases associated with defects in lysosomal storage |
| GBGB1702551.1A GB201702551D0 (en) | 2017-02-16 | 2017-02-16 | Therapeutic agents for neurodegenerative diseases, lysosomal storage disorders and neurodegenerative diseases associated with defects in lysosomal storage |
| GB1702551.1 | 2017-02-16 | ||
| GB1705766.2 | 2017-04-10 | ||
| GBGB1705766.2A GB201705766D0 (en) | 2017-04-10 | 2017-04-10 | Therapeutic agents for neurodegenerative diseases, lysosomal storage disorders and neurodegenerative diseases associated with defects in lysosomal storage |
| GB1706867.7 | 2017-04-28 | ||
| GBGB1706867.7A GB201706867D0 (en) | 2017-04-28 | 2017-04-28 | Therapeutic agents for neurodegenerative diseases, lysosomal storage disorders and neurodegenerative diseases associated with defects in lysosomal storage |
| KR1020227021012A KR20220093385A (ko) | 2016-08-11 | 2017-08-11 | 신경퇴행성 질병을 위한 치료제 |
| PCT/IB2017/054929 WO2018029658A1 (en) | 2016-08-11 | 2017-08-11 | Therapeutic agents for neurodegenerative diseases |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020227021012A Division KR20220093385A (ko) | 2016-08-11 | 2017-08-11 | 신경퇴행성 질병을 위한 치료제 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020257008819A Division KR20250042197A (ko) | 2016-08-11 | 2017-08-11 | 신경퇴행성 질병을 위한 치료제 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| KR20240068761A KR20240068761A (ko) | 2024-05-17 |
| KR102785448B1 true KR102785448B1 (ko) | 2025-03-21 |
Family
ID=59859424
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020247014635A Active KR102785448B1 (ko) | 2016-08-11 | 2017-08-11 | 신경퇴행성 질병을 위한 치료제 |
| KR1020257008819A Pending KR20250042197A (ko) | 2016-08-11 | 2017-08-11 | 신경퇴행성 질병을 위한 치료제 |
| KR1020197007032A Active KR102413756B1 (ko) | 2016-08-11 | 2017-08-11 | 신경퇴행성 질병을 위한 치료제 |
| KR1020227021012A Ceased KR20220093385A (ko) | 2016-08-11 | 2017-08-11 | 신경퇴행성 질병을 위한 치료제 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020257008819A Pending KR20250042197A (ko) | 2016-08-11 | 2017-08-11 | 신경퇴행성 질병을 위한 치료제 |
| KR1020197007032A Active KR102413756B1 (ko) | 2016-08-11 | 2017-08-11 | 신경퇴행성 질병을 위한 치료제 |
| KR1020227021012A Ceased KR20220093385A (ko) | 2016-08-11 | 2017-08-11 | 신경퇴행성 질병을 위한 치료제 |
Country Status (28)
| Country | Link |
|---|---|
| US (3) | US12144792B2 (enExample) |
| EP (3) | EP3416631B1 (enExample) |
| JP (3) | JP6957602B2 (enExample) |
| KR (4) | KR102785448B1 (enExample) |
| CN (4) | CN121081445A (enExample) |
| AU (3) | AU2017308865B2 (enExample) |
| BR (1) | BR112019002730A2 (enExample) |
| CA (1) | CA3033564A1 (enExample) |
| CY (1) | CY1121930T1 (enExample) |
| DK (1) | DK3416631T3 (enExample) |
| ES (1) | ES2733677T3 (enExample) |
| HR (1) | HRP20191055T1 (enExample) |
| HU (1) | HUE045043T2 (enExample) |
| IL (3) | IL310801A (enExample) |
| LT (1) | LT3416631T (enExample) |
| MA (2) | MA47521A (enExample) |
| MD (1) | MD3416631T2 (enExample) |
| ME (1) | ME03454B (enExample) |
| MX (3) | MX383499B (enExample) |
| PL (1) | PL3416631T3 (enExample) |
| PT (1) | PT3416631T (enExample) |
| RS (1) | RS59048B1 (enExample) |
| RU (1) | RU2756519C2 (enExample) |
| SG (1) | SG11201901063SA (enExample) |
| SI (1) | SI3416631T1 (enExample) |
| SM (1) | SMT201900370T1 (enExample) |
| TN (1) | TN2019000033A1 (enExample) |
| WO (1) | WO2018029658A1 (enExample) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10219815B2 (en) | 2005-09-22 | 2019-03-05 | The Regents Of The University Of Michigan | Histotripsy for thrombolysis |
| ES2948135T3 (es) | 2015-06-24 | 2023-08-31 | Univ Michigan Regents | Sistemas de terapia de histotripsia para el tratamiento del tejido cerebral |
| PL3416631T3 (pl) | 2016-08-11 | 2019-11-29 | Intrabio Ltd | Środki terapeutyczne do leczenia chorób neurodegeneracyjnych |
| MA55947A (fr) * | 2016-08-11 | 2022-03-23 | Intrabio Ltd | Compositions pharmaceutiques et utilisations dirigées vers des troubles d'entreposage lysosomial |
| GB201709459D0 (en) | 2017-06-14 | 2017-07-26 | Intrabio Ltd | Treatment for migraine |
| CN111542314B (zh) * | 2017-10-18 | 2023-11-24 | 内在生物技术有限公司 | 神经退行性疾病的治疗剂 |
| CA3091353A1 (en) | 2018-02-15 | 2019-08-22 | Intrabio Ltd | Therapeutic agents for treating restless legs syndrome |
| CN120324803A (zh) | 2018-11-28 | 2025-07-18 | 希斯托索尼克斯公司 | 组织摧毁术系统及方法 |
| WO2020115715A1 (en) * | 2018-12-06 | 2020-06-11 | Intrabio Ltd. | Deuterated analogs of acetyl-leucine |
| IL321899A (en) | 2019-03-02 | 2025-09-01 | Intrabio Ltd | Leucine, acetyl leucine and related analogs for the treatment of disease |
| US20220346699A1 (en) * | 2019-09-17 | 2022-11-03 | Hoffmann-La Roche Inc. | Improvements in Personalized Healthcare for Patients with Movement Disorders |
| US20230051742A1 (en) * | 2020-01-13 | 2023-02-16 | Intrabio Ltd. | Treatment of late-onset neurodegenerative diseases in heterozygous npc1 gene mutation carriers |
| WO2022264037A1 (en) * | 2021-06-14 | 2022-12-22 | Intrabio Ltd. | Branched-chain amino acid derivatives to treat disease |
| EP4608504A1 (en) | 2022-10-28 | 2025-09-03 | Histosonics, Inc. | Histotripsy systems and methods |
| AU2024257180A1 (en) | 2023-04-20 | 2025-09-18 | Histosonics, Inc. | Histotripsy systems and associated methods including user interfaces and workflows for treatment planning and therapy |
| WO2025151578A1 (en) * | 2024-01-12 | 2025-07-17 | Intrabio Inc. | N-acetyl-leucine for use in the treatment of prodromal alpha-synucleinopathies |
| WO2025163129A1 (en) * | 2024-02-01 | 2025-08-07 | Intrabio Ltd. | Acetyl-leucine for treating parkinson´s disease |
| US12403111B2 (en) | 2024-02-02 | 2025-09-02 | SynGAP Research Fund | Compositions and methods for treating neurodevelopmental disorders |
Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2011151685A1 (en) | 2010-06-03 | 2011-12-08 | Raouf Rekik | N-acetyl-dl-leucine, neuroprotective and retinoprotective medicament |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20060063827A1 (en) | 2004-09-23 | 2006-03-23 | Yu Ruey J | Systemic administration of therapeutic amino acids and N-acetylamino acids |
| US20050222209A1 (en) * | 2004-04-01 | 2005-10-06 | Zeldis Jerome B | Methods and compositions for the treatment, prevention or management of dysfunctional sleep and dysfunctional sleep associated with disease |
| FR2905600B1 (fr) * | 2006-09-13 | 2010-01-15 | Pf Medicament | Traitement des vertiges par l'acetyl-l-leucine. |
| EP2946779A1 (en) | 2007-02-22 | 2015-11-25 | Beiersdorf AG | Cosmetic and pharmaceutical applications of n-acetylhydroxyproline |
| TN2010000566A1 (en) | 2010-12-03 | 2012-05-24 | Rekik Raouf | Folic acid - ramipril combination : cell protective neuroprotective and retinoprotective ophtalmologic drugs |
| EP3062810A4 (en) * | 2013-10-28 | 2017-05-03 | Naurex Inc. | Nmda receptor modulators and prodrugs, salts, and uses thereof |
| KR102413754B1 (ko) | 2016-04-19 | 2022-06-27 | 인트라바이오 리미티드 | 이동성 및 인지 기능을 개선시키기 위한 아세틸-류신 또는 이의 약제학적으로 허용되는 염 |
| PL3416631T3 (pl) | 2016-08-11 | 2019-11-29 | Intrabio Ltd | Środki terapeutyczne do leczenia chorób neurodegeneracyjnych |
-
2017
- 2017-08-11 PL PL17767934T patent/PL3416631T3/pl unknown
- 2017-08-11 MA MA047521A patent/MA47521A/fr unknown
- 2017-08-11 SG SG11201901063SA patent/SG11201901063SA/en unknown
- 2017-08-11 KR KR1020247014635A patent/KR102785448B1/ko active Active
- 2017-08-11 TN TNP/2019/000033A patent/TN2019000033A1/fr unknown
- 2017-08-11 CN CN202511449973.8A patent/CN121081445A/zh active Pending
- 2017-08-11 KR KR1020257008819A patent/KR20250042197A/ko active Pending
- 2017-08-11 IL IL310801A patent/IL310801A/en unknown
- 2017-08-11 IL IL310799A patent/IL310799A/en unknown
- 2017-08-11 CA CA3033564A patent/CA3033564A1/en active Pending
- 2017-08-11 EP EP17767934.7A patent/EP3416631B1/en active Active
- 2017-08-11 RS RS20190801A patent/RS59048B1/sr unknown
- 2017-08-11 SM SM20190370T patent/SMT201900370T1/it unknown
- 2017-08-11 IL IL264641A patent/IL264641B2/en unknown
- 2017-08-11 DK DK17767934.7T patent/DK3416631T3/da active
- 2017-08-11 ME MEP-2019-169A patent/ME03454B/me unknown
- 2017-08-11 BR BR112019002730-0A patent/BR112019002730A2/pt not_active Application Discontinuation
- 2017-08-11 SI SI201730071T patent/SI3416631T1/sl unknown
- 2017-08-11 HU HUE17767934A patent/HUE045043T2/hu unknown
- 2017-08-11 CN CN202310627214.0A patent/CN116459244A/zh active Pending
- 2017-08-11 EP EP19174007.5A patent/EP3583940A1/en active Pending
- 2017-08-11 MX MX2019001575A patent/MX383499B/es unknown
- 2017-08-11 US US16/324,353 patent/US12144792B2/en active Active
- 2017-08-11 MA MA43828A patent/MA43828B1/fr unknown
- 2017-08-11 AU AU2017308865A patent/AU2017308865B2/en active Active
- 2017-08-11 JP JP2019507819A patent/JP6957602B2/ja active Active
- 2017-08-11 CN CN202310629563.6A patent/CN116492328A/zh active Pending
- 2017-08-11 WO PCT/IB2017/054929 patent/WO2018029658A1/en not_active Ceased
- 2017-08-11 ES ES17767934T patent/ES2733677T3/es active Active
- 2017-08-11 LT LTEP17767934.7T patent/LT3416631T/lt unknown
- 2017-08-11 KR KR1020197007032A patent/KR102413756B1/ko active Active
- 2017-08-11 HR HRP20191055TT patent/HRP20191055T1/hr unknown
- 2017-08-11 PT PT17767934T patent/PT3416631T/pt unknown
- 2017-08-11 CN CN201780059708.6A patent/CN109789114A/zh active Pending
- 2017-08-11 KR KR1020227021012A patent/KR20220093385A/ko not_active Ceased
- 2017-08-11 RU RU2019106506A patent/RU2756519C2/ru active
- 2017-08-11 EP EP24191483.7A patent/EP4467195A3/en active Pending
- 2017-08-11 MD MDE20190027T patent/MD3416631T2/ro unknown
-
2019
- 2019-02-07 MX MX2021006901A patent/MX2021006901A/es unknown
- 2019-07-16 CY CY20191100759T patent/CY1121930T1/el unknown
-
2021
- 2021-10-06 JP JP2021164793A patent/JP2022003085A/ja active Pending
-
2023
- 2023-03-15 US US18/183,972 patent/US20230210799A1/en active Pending
- 2023-04-05 AU AU2023202121A patent/AU2023202121B2/en active Active
- 2023-09-08 JP JP2023146465A patent/JP2023175762A/ja active Pending
-
2024
- 2024-01-12 US US18/411,512 patent/US20240189267A1/en active Pending
- 2024-04-11 MX MX2024004439A patent/MX2024004439A/es unknown
-
2025
- 2025-03-21 AU AU2025202077A patent/AU2025202077A1/en active Pending
Patent Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2011151685A1 (en) | 2010-06-03 | 2011-12-08 | Raouf Rekik | N-acetyl-dl-leucine, neuroprotective and retinoprotective medicament |
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| KR102785448B1 (ko) | 신경퇴행성 질병을 위한 치료제 | |
| KR102549684B1 (ko) | 신경퇴행성 질병에 대한 치료제 | |
| RU2849887C2 (ru) | Терапевтические средства против нейродегенеративных заболеваний | |
| TW202539621A (zh) | 用於治療巴金森氏症的治療劑 | |
| HK40112206A (en) | Therapeutic agents for neurodegenerative diseases | |
| HK40018125A (en) | Therapeutic agents for neurodegenerative diseases | |
| NZ790893A (en) | Therapeutic Agents for Neurodegenerative Diseases | |
| HK1259779B (en) | Therapeutic agents for neurodegenerative diseases | |
| HK1259779A1 (en) | Therapeutic agents for neurodegenerative diseases |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A107 | Divisional application of patent | ||
| PA0104 | Divisional application for international application |
Comment text: Divisional Application for International Patent Patent event code: PA01041R01D Patent event date: 20240430 Application number text: 1020227021012 Filing date: 20220621 |
|
| PA0201 | Request for examination | ||
| PG1501 | Laying open of application | ||
| E902 | Notification of reason for refusal | ||
| PE0902 | Notice of grounds for rejection |
Comment text: Notification of reason for refusal Patent event date: 20241002 Patent event code: PE09021S01D |
|
| E701 | Decision to grant or registration of patent right | ||
| PE0701 | Decision of registration |
Patent event code: PE07011S01D Comment text: Decision to Grant Registration Patent event date: 20250203 |
|
| A107 | Divisional application of patent | ||
| PA0104 | Divisional application for international application |
Comment text: Divisional Application for International Patent Patent event code: PA01041R01D Patent event date: 20250318 Application number text: 1020227021012 Filing date: 20220621 |
|
| GRNT | Written decision to grant | ||
| PR0701 | Registration of establishment |
Comment text: Registration of Establishment Patent event date: 20250319 Patent event code: PR07011E01D |
|
| PR1002 | Payment of registration fee |
Payment date: 20250319 End annual number: 3 Start annual number: 1 |
|
| PG1601 | Publication of registration |