KR20230092905A - NF-κB-매개 질환의 치료 - Google Patents
NF-κB-매개 질환의 치료 Download PDFInfo
- Publication number
- KR20230092905A KR20230092905A KR1020237013258A KR20237013258A KR20230092905A KR 20230092905 A KR20230092905 A KR 20230092905A KR 1020237013258 A KR1020237013258 A KR 1020237013258A KR 20237013258 A KR20237013258 A KR 20237013258A KR 20230092905 A KR20230092905 A KR 20230092905A
- Authority
- KR
- South Korea
- Prior art keywords
- day
- human patient
- muscular dystrophy
- participants
- age
- 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.)
- Pending
Links
- 238000011282 treatment Methods 0.000 title claims description 99
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title description 53
- 201000010099 disease Diseases 0.000 title description 42
- 108010057466 NF-kappa B Proteins 0.000 title description 16
- 102000003945 NF-kappa B Human genes 0.000 title description 16
- 230000001404 mediated effect Effects 0.000 title description 10
- 238000000034 method Methods 0.000 claims abstract description 61
- 208000024891 symptom Diseases 0.000 claims abstract description 36
- 201000006938 muscular dystrophy Diseases 0.000 claims abstract description 33
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims description 62
- 229960004618 prednisone Drugs 0.000 claims description 57
- XOFYZVNMUHMLCC-ZPOLXVRWSA-N prednisone Chemical compound O=C1C=C[C@]2(C)[C@H]3C(=O)C[C@](C)([C@@](CC4)(O)C(=O)CO)[C@@H]4[C@@H]3CCC2=C1 XOFYZVNMUHMLCC-ZPOLXVRWSA-N 0.000 claims description 57
- 150000001875 compounds Chemical class 0.000 claims description 44
- 239000000203 mixture Substances 0.000 claims description 36
- FBHSPRKOSMHSIF-GRMWVWQJSA-N deflazacort Chemical compound C1CC2=CC(=O)C=C[C@]2(C)[C@@H]2[C@@H]1[C@@H]1C[C@H]3OC(C)=N[C@@]3(C(=O)COC(=O)C)[C@@]1(C)C[C@@H]2O FBHSPRKOSMHSIF-GRMWVWQJSA-N 0.000 claims description 32
- 229960001145 deflazacort Drugs 0.000 claims description 27
- 210000000988 bone and bone Anatomy 0.000 claims description 25
- 230000008859 change Effects 0.000 claims description 23
- 230000002411 adverse Effects 0.000 claims description 17
- 239000000725 suspension Substances 0.000 claims description 17
- 230000003542 behavioural effect Effects 0.000 claims description 15
- 230000002829 reductive effect Effects 0.000 claims description 15
- 208000021017 Weight Gain Diseases 0.000 claims description 13
- 230000004584 weight gain Effects 0.000 claims description 13
- 235000019786 weight gain Nutrition 0.000 claims description 13
- 208000019901 Anxiety disease Diseases 0.000 claims description 8
- 206010034719 Personality change Diseases 0.000 claims description 7
- 206010027951 Mood swings Diseases 0.000 claims description 6
- 230000036506 anxiety Effects 0.000 claims description 6
- 229910052500 inorganic mineral Inorganic materials 0.000 claims description 6
- 239000011707 mineral Substances 0.000 claims description 6
- 230000002103 transcriptional effect Effects 0.000 claims description 5
- 206010022998 Irritability Diseases 0.000 claims description 4
- 238000013019 agitation Methods 0.000 claims description 4
- 230000001186 cumulative effect Effects 0.000 claims description 4
- 238000009547 dual-energy X-ray absorptiometry Methods 0.000 claims description 4
- 208000019116 sleep disease Diseases 0.000 claims description 4
- 206010041569 spinal fracture Diseases 0.000 claims description 4
- 208000013738 Sleep Initiation and Maintenance disease Diseases 0.000 claims description 3
- 230000016571 aggressive behavior Effects 0.000 claims description 3
- 230000037182 bone density Effects 0.000 claims description 3
- 230000004970 emotional disturbance Effects 0.000 claims description 3
- 206010022437 insomnia Diseases 0.000 claims description 3
- 208000022925 sleep disturbance Diseases 0.000 claims description 3
- 208000001132 Osteoporosis Diseases 0.000 claims description 2
- 239000003205 fragrance Substances 0.000 claims 1
- 150000003839 salts Chemical class 0.000 abstract description 16
- 239000003246 corticosteroid Substances 0.000 description 108
- ZYTXTXAMMDTYDQ-DGEXFFLYSA-N vamorolone Chemical compound C([C@H]12)CC3=CC(=O)C=C[C@]3(C)C1=CC[C@@]1(C)[C@H]2C[C@@H](C)[C@]1(O)C(=O)CO ZYTXTXAMMDTYDQ-DGEXFFLYSA-N 0.000 description 46
- 229960001334 corticosteroids Drugs 0.000 description 41
- 229950011597 vamorolone Drugs 0.000 description 40
- 230000012010 growth Effects 0.000 description 30
- 230000006872 improvement Effects 0.000 description 22
- 238000004448 titration Methods 0.000 description 18
- 229940079593 drug Drugs 0.000 description 17
- 239000003814 drug Substances 0.000 description 17
- 230000000694 effects Effects 0.000 description 17
- 238000012423 maintenance Methods 0.000 description 16
- 239000000243 solution Substances 0.000 description 16
- 238000004458 analytical method Methods 0.000 description 14
- OKKJLVBELUTLKV-UHFFFAOYSA-N Methanol Chemical compound OC OKKJLVBELUTLKV-UHFFFAOYSA-N 0.000 description 12
- 230000007774 longterm Effects 0.000 description 11
- 239000007787 solid Substances 0.000 description 11
- WYURNTSHIVDZCO-UHFFFAOYSA-N Tetrahydrofuran Chemical compound C1CCOC1 WYURNTSHIVDZCO-UHFFFAOYSA-N 0.000 description 10
- 238000004128 high performance liquid chromatography Methods 0.000 description 10
- QTBSBXVTEAMEQO-UHFFFAOYSA-N Acetic acid Chemical compound CC(O)=O QTBSBXVTEAMEQO-UHFFFAOYSA-N 0.000 description 9
- YMWUJEATGCHHMB-UHFFFAOYSA-N Dichloromethane Chemical compound ClCCl YMWUJEATGCHHMB-UHFFFAOYSA-N 0.000 description 9
- 230000007423 decrease Effects 0.000 description 9
- 208000035475 disorder Diseases 0.000 description 9
- 230000009036 growth inhibition Effects 0.000 description 9
- 230000017066 negative regulation of growth Effects 0.000 description 9
- 229940068196 placebo Drugs 0.000 description 9
- 239000000902 placebo Substances 0.000 description 9
- 238000000634 powder X-ray diffraction Methods 0.000 description 9
- 239000000047 product Substances 0.000 description 9
- 230000008901 benefit Effects 0.000 description 8
- 230000001684 chronic effect Effects 0.000 description 8
- 238000002560 therapeutic procedure Methods 0.000 description 8
- 238000011156 evaluation Methods 0.000 description 7
- 230000004044 response Effects 0.000 description 7
- YXFVVABEGXRONW-UHFFFAOYSA-N Toluene Chemical compound CC1=CC=CC=C1 YXFVVABEGXRONW-UHFFFAOYSA-N 0.000 description 6
- 239000012535 impurity Substances 0.000 description 6
- 238000004519 manufacturing process Methods 0.000 description 6
- 238000002360 preparation method Methods 0.000 description 6
- 230000002265 prevention Effects 0.000 description 6
- 238000007619 statistical method Methods 0.000 description 6
- 238000012360 testing method Methods 0.000 description 6
- 206010053759 Growth retardation Diseases 0.000 description 5
- 206010028289 Muscle atrophy Diseases 0.000 description 5
- 239000013543 active substance Substances 0.000 description 5
- 239000000090 biomarker Substances 0.000 description 5
- 150000002118 epoxides Chemical class 0.000 description 5
- 231100000001 growth retardation Toxicity 0.000 description 5
- 201000000585 muscular atrophy Diseases 0.000 description 5
- 239000012044 organic layer Substances 0.000 description 5
- 238000011084 recovery Methods 0.000 description 5
- XLYOFNOQVPJJNP-UHFFFAOYSA-N water Substances O XLYOFNOQVPJJNP-UHFFFAOYSA-N 0.000 description 5
- IJGRMHOSHXDMSA-UHFFFAOYSA-N Atomic nitrogen Chemical compound N#N IJGRMHOSHXDMSA-UHFFFAOYSA-N 0.000 description 4
- 208000017667 Chronic Disease Diseases 0.000 description 4
- 206010020112 Hirsutism Diseases 0.000 description 4
- 241001465754 Metazoa Species 0.000 description 4
- 239000012267 brine Substances 0.000 description 4
- 238000006243 chemical reaction Methods 0.000 description 4
- 230000003247 decreasing effect Effects 0.000 description 4
- 238000004090 dissolution Methods 0.000 description 4
- GUVUOGQBMYCBQP-UHFFFAOYSA-N dmpu Chemical compound CN1CCCN(C)C1=O GUVUOGQBMYCBQP-UHFFFAOYSA-N 0.000 description 4
- 230000036541 health Effects 0.000 description 4
- 230000002757 inflammatory effect Effects 0.000 description 4
- 235000010755 mineral Nutrition 0.000 description 4
- 238000012986 modification Methods 0.000 description 4
- 230000004048 modification Effects 0.000 description 4
- 238000012544 monitoring process Methods 0.000 description 4
- 230000007659 motor function Effects 0.000 description 4
- 210000003205 muscle Anatomy 0.000 description 4
- 238000011160 research Methods 0.000 description 4
- HPALAKNZSZLMCH-UHFFFAOYSA-M sodium;chloride;hydrate Chemical compound O.[Na+].[Cl-] HPALAKNZSZLMCH-UHFFFAOYSA-M 0.000 description 4
- 238000013517 stratification Methods 0.000 description 4
- WEVYAHXRMPXWCK-UHFFFAOYSA-N Acetonitrile Chemical compound CC#N WEVYAHXRMPXWCK-UHFFFAOYSA-N 0.000 description 3
- 208000002177 Cataract Diseases 0.000 description 3
- 208000011688 Generalised anxiety disease Diseases 0.000 description 3
- 102000003676 Glucocorticoid Receptors Human genes 0.000 description 3
- 108090000079 Glucocorticoid Receptors Proteins 0.000 description 3
- 241000282412 Homo Species 0.000 description 3
- 108090000375 Mineralocorticoid Receptors Proteins 0.000 description 3
- 208000000810 Separation Anxiety Diseases 0.000 description 3
- 206010041250 Social phobia Diseases 0.000 description 3
- UIIMBOGNXHQVGW-UHFFFAOYSA-M Sodium bicarbonate Chemical compound [Na+].OC([O-])=O UIIMBOGNXHQVGW-UHFFFAOYSA-M 0.000 description 3
- 239000004480 active ingredient Substances 0.000 description 3
- 230000003110 anti-inflammatory effect Effects 0.000 description 3
- 230000027455 binding Effects 0.000 description 3
- 239000008280 blood Substances 0.000 description 3
- 210000004369 blood Anatomy 0.000 description 3
- 230000037396 body weight Effects 0.000 description 3
- 230000006378 damage Effects 0.000 description 3
- 238000011161 development Methods 0.000 description 3
- 230000018109 developmental process Effects 0.000 description 3
- 239000000706 filtrate Substances 0.000 description 3
- 230000006870 function Effects 0.000 description 3
- 208000029364 generalized anxiety disease Diseases 0.000 description 3
- 239000003862 glucocorticoid Substances 0.000 description 3
- 239000004615 ingredient Substances 0.000 description 3
- 239000010410 layer Substances 0.000 description 3
- 239000000463 material Substances 0.000 description 3
- 230000002232 neuromuscular Effects 0.000 description 3
- 239000008203 oral pharmaceutical composition Substances 0.000 description 3
- 230000037361 pathway Effects 0.000 description 3
- 239000007971 pharmaceutical suspension Substances 0.000 description 3
- 230000000704 physical effect Effects 0.000 description 3
- 230000034190 positive regulation of NF-kappaB transcription factor activity Effects 0.000 description 3
- 108090000623 proteins and genes Proteins 0.000 description 3
- 208000020016 psychiatric disease Diseases 0.000 description 3
- 230000009467 reduction Effects 0.000 description 3
- 208000025874 separation anxiety disease Diseases 0.000 description 3
- 210000002027 skeletal muscle Anatomy 0.000 description 3
- 239000002904 solvent Substances 0.000 description 3
- 150000003431 steroids Chemical class 0.000 description 3
- 239000000126 substance Substances 0.000 description 3
- 238000013518 transcription Methods 0.000 description 3
- 230000035897 transcription Effects 0.000 description 3
- 206010000117 Abnormal behaviour Diseases 0.000 description 2
- 208000032170 Congenital Abnormalities Diseases 0.000 description 2
- 206010061818 Disease progression Diseases 0.000 description 2
- 201000009273 Endometriosis Diseases 0.000 description 2
- XEKOWRVHYACXOJ-UHFFFAOYSA-N Ethyl acetate Chemical compound CCOC(C)=O XEKOWRVHYACXOJ-UHFFFAOYSA-N 0.000 description 2
- 206010017076 Fracture Diseases 0.000 description 2
- 208000010412 Glaucoma Diseases 0.000 description 2
- 206010020864 Hypertrichosis Diseases 0.000 description 2
- 208000019693 Lung disease Diseases 0.000 description 2
- 102000003979 Mineralocorticoid Receptors Human genes 0.000 description 2
- 208000019022 Mood disease Diseases 0.000 description 2
- IMNFDUFMRHMDMM-UHFFFAOYSA-N N-Heptane Chemical compound CCCCCCC IMNFDUFMRHMDMM-UHFFFAOYSA-N 0.000 description 2
- 206010028980 Neoplasm Diseases 0.000 description 2
- KFSLWBXXFJQRDL-UHFFFAOYSA-N Peracetic acid Chemical compound CC(=O)OO KFSLWBXXFJQRDL-UHFFFAOYSA-N 0.000 description 2
- VYPSYNLAJGMNEJ-UHFFFAOYSA-N Silicium dioxide Chemical compound O=[Si]=O VYPSYNLAJGMNEJ-UHFFFAOYSA-N 0.000 description 2
- DTQVDTLACAAQTR-UHFFFAOYSA-N Trifluoroacetic acid Chemical compound OC(=O)C(F)(F)F DTQVDTLACAAQTR-UHFFFAOYSA-N 0.000 description 2
- 208000027418 Wounds and injury Diseases 0.000 description 2
- 230000002159 abnormal effect Effects 0.000 description 2
- 238000010521 absorption reaction Methods 0.000 description 2
- 239000012190 activator Substances 0.000 description 2
- 208000026935 allergic disease Diseases 0.000 description 2
- 239000005557 antagonist Substances 0.000 description 2
- 229940124599 anti-inflammatory drug Drugs 0.000 description 2
- 208000006673 asthma Diseases 0.000 description 2
- 230000006399 behavior Effects 0.000 description 2
- 231100000871 behavioral problem Toxicity 0.000 description 2
- 244000309466 calf Species 0.000 description 2
- 230000000052 comparative effect Effects 0.000 description 2
- 229940125782 compound 2 Drugs 0.000 description 2
- 229940126214 compound 3 Drugs 0.000 description 2
- OPQARKPSCNTWTJ-UHFFFAOYSA-L copper(ii) acetate Chemical compound [Cu+2].CC([O-])=O.CC([O-])=O OPQARKPSCNTWTJ-UHFFFAOYSA-L 0.000 description 2
- 239000013078 crystal Substances 0.000 description 2
- 230000003111 delayed effect Effects 0.000 description 2
- 206010012601 diabetes mellitus Diseases 0.000 description 2
- 238000010586 diagram Methods 0.000 description 2
- 230000006806 disease prevention Effects 0.000 description 2
- 231100000673 dose–response relationship Toxicity 0.000 description 2
- -1 epimers Substances 0.000 description 2
- 239000006260 foam Substances 0.000 description 2
- 230000009760 functional impairment Effects 0.000 description 2
- 230000002068 genetic effect Effects 0.000 description 2
- JYGXADMDTFJGBT-VWUMJDOOSA-N hydrocortisone Chemical compound O=C1CC[C@]2(C)[C@H]3[C@@H](O)C[C@](C)([C@@](CC4)(O)C(=O)CO)[C@@H]4[C@@H]3CCC2=C1 JYGXADMDTFJGBT-VWUMJDOOSA-N 0.000 description 2
- RAXXELZNTBOGNW-UHFFFAOYSA-N imidazole Substances C1=CNC=N1 RAXXELZNTBOGNW-UHFFFAOYSA-N 0.000 description 2
- 206010021654 increased appetite Diseases 0.000 description 2
- 208000015181 infectious disease Diseases 0.000 description 2
- 230000002401 inhibitory effect Effects 0.000 description 2
- 230000005764 inhibitory process Effects 0.000 description 2
- 230000000977 initiatory effect Effects 0.000 description 2
- 208000014674 injury Diseases 0.000 description 2
- 208000017169 kidney disease Diseases 0.000 description 2
- 230000000670 limiting effect Effects 0.000 description 2
- 239000007788 liquid Substances 0.000 description 2
- 238000011866 long-term treatment Methods 0.000 description 2
- CCERQOYLJJULMD-UHFFFAOYSA-M magnesium;carbanide;chloride Chemical compound [CH3-].[Mg+2].[Cl-] CCERQOYLJJULMD-UHFFFAOYSA-M 0.000 description 2
- 231100000682 maximum tolerated dose Toxicity 0.000 description 2
- 238000005259 measurement Methods 0.000 description 2
- 230000020763 muscle atrophy Effects 0.000 description 2
- 229910052757 nitrogen Inorganic materials 0.000 description 2
- 238000010831 paired-sample T-test Methods 0.000 description 2
- 239000002245 particle Substances 0.000 description 2
- 238000003921 particle size analysis Methods 0.000 description 2
- 230000003389 potentiating effect Effects 0.000 description 2
- 229960005205 prednisolone Drugs 0.000 description 2
- OIGNJSKKLXVSLS-VWUMJDOOSA-N prednisolone Chemical compound O=C1C=C[C@]2(C)[C@H]3[C@@H](O)C[C@](C)([C@@](CC4)(O)C(=O)CO)[C@@H]4[C@@H]3CCC2=C1 OIGNJSKKLXVSLS-VWUMJDOOSA-N 0.000 description 2
- 125000002324 prednisone group Chemical group 0.000 description 2
- 238000012545 processing Methods 0.000 description 2
- 230000000750 progressive effect Effects 0.000 description 2
- 102000004169 proteins and genes Human genes 0.000 description 2
- 238000001953 recrystallisation Methods 0.000 description 2
- 230000001105 regulatory effect Effects 0.000 description 2
- 238000004626 scanning electron microscopy Methods 0.000 description 2
- 239000002002 slurry Substances 0.000 description 2
- 239000011734 sodium Substances 0.000 description 2
- 239000007858 starting material Substances 0.000 description 2
- 238000003756 stirring Methods 0.000 description 2
- 238000003860 storage Methods 0.000 description 2
- YLQBMQCUIZJEEH-UHFFFAOYSA-N tetrahydrofuran Natural products C=1C=COC=1 YLQBMQCUIZJEEH-UHFFFAOYSA-N 0.000 description 2
- 230000001225 therapeutic effect Effects 0.000 description 2
- 210000001519 tissue Anatomy 0.000 description 2
- 231100000419 toxicity Toxicity 0.000 description 2
- 230000001988 toxicity Effects 0.000 description 2
- 238000005406 washing Methods 0.000 description 2
- 208000030507 AIDS Diseases 0.000 description 1
- 208000022309 Alcoholic Liver disease Diseases 0.000 description 1
- 208000024827 Alzheimer disease Diseases 0.000 description 1
- 206010002383 Angina Pectoris Diseases 0.000 description 1
- 208000003343 Antiphospholipid Syndrome Diseases 0.000 description 1
- 206010002869 Anxiety symptoms Diseases 0.000 description 1
- 206010003011 Appendicitis Diseases 0.000 description 1
- 206010003591 Ataxia Diseases 0.000 description 1
- 201000001320 Atherosclerosis Diseases 0.000 description 1
- 208000023275 Autoimmune disease Diseases 0.000 description 1
- 201000006935 Becker muscular dystrophy Diseases 0.000 description 1
- 208000009137 Behcet syndrome Diseases 0.000 description 1
- 208000010392 Bone Fractures Diseases 0.000 description 1
- 206010065687 Bone loss Diseases 0.000 description 1
- 206010007558 Cardiac failure chronic Diseases 0.000 description 1
- 206010007572 Cardiac hypertrophy Diseases 0.000 description 1
- 208000006029 Cardiomegaly Diseases 0.000 description 1
- 208000031229 Cardiomyopathies Diseases 0.000 description 1
- 208000006545 Chronic Obstructive Pulmonary Disease Diseases 0.000 description 1
- 208000023890 Complex Regional Pain Syndromes Diseases 0.000 description 1
- 208000011231 Crohn disease Diseases 0.000 description 1
- 201000003883 Cystic fibrosis Diseases 0.000 description 1
- 102000004127 Cytokines Human genes 0.000 description 1
- 108090000695 Cytokines Proteins 0.000 description 1
- 108020004414 DNA Proteins 0.000 description 1
- 102000004163 DNA-directed RNA polymerases Human genes 0.000 description 1
- 108090000626 DNA-directed RNA polymerases Proteins 0.000 description 1
- 206010011878 Deafness Diseases 0.000 description 1
- 102000001039 Dystrophin Human genes 0.000 description 1
- 108010069091 Dystrophin Proteins 0.000 description 1
- 206010016207 Familial Mediterranean fever Diseases 0.000 description 1
- 208000001362 Fetal Growth Retardation Diseases 0.000 description 1
- 206010070531 Foetal growth restriction Diseases 0.000 description 1
- 206010017577 Gait disturbance Diseases 0.000 description 1
- 208000022461 Glomerular disease Diseases 0.000 description 1
- 206010018429 Glucose tolerance impaired Diseases 0.000 description 1
- 206010019233 Headaches Diseases 0.000 description 1
- 206010019280 Heart failures Diseases 0.000 description 1
- 241000713772 Human immunodeficiency virus 1 Species 0.000 description 1
- 208000023105 Huntington disease Diseases 0.000 description 1
- 208000035150 Hypercholesterolemia Diseases 0.000 description 1
- 208000008454 Hyperhidrosis Diseases 0.000 description 1
- 208000029084 Hyperlordosis Diseases 0.000 description 1
- 206010020751 Hypersensitivity Diseases 0.000 description 1
- 206010020772 Hypertension Diseases 0.000 description 1
- 206010020850 Hyperthyroidism Diseases 0.000 description 1
- 206010020880 Hypertrophy Diseases 0.000 description 1
- 206010058359 Hypogonadism Diseases 0.000 description 1
- 238000004566 IR spectroscopy Methods 0.000 description 1
- 206010062016 Immunosuppression Diseases 0.000 description 1
- 208000022559 Inflammatory bowel disease Diseases 0.000 description 1
- 201000008450 Intracranial aneurysm Diseases 0.000 description 1
- 208000034693 Laceration Diseases 0.000 description 1
- 102100022745 Laminin subunit alpha-2 Human genes 0.000 description 1
- 206010024238 Leptospirosis Diseases 0.000 description 1
- 208000004852 Lung Injury Diseases 0.000 description 1
- 208000005777 Lupus Nephritis Diseases 0.000 description 1
- 241000124008 Mammalia Species 0.000 description 1
- 206010027406 Mesothelioma Diseases 0.000 description 1
- 208000029725 Metabolic bone disease Diseases 0.000 description 1
- 102100021316 Mineralocorticoid receptor Human genes 0.000 description 1
- 206010028311 Muscle hypertrophy Diseases 0.000 description 1
- 208000023178 Musculoskeletal disease Diseases 0.000 description 1
- 206010052904 Musculoskeletal stiffness Diseases 0.000 description 1
- 208000000112 Myalgia Diseases 0.000 description 1
- 206010028629 Myoglobinuria Diseases 0.000 description 1
- YKFRUJSEPGHZFJ-UHFFFAOYSA-N N-trimethylsilylimidazole Chemical compound C[Si](C)(C)N1C=CN=C1 YKFRUJSEPGHZFJ-UHFFFAOYSA-N 0.000 description 1
- 208000008589 Obesity Diseases 0.000 description 1
- 201000009110 Oculopharyngeal muscular dystrophy Diseases 0.000 description 1
- 208000010191 Osteitis Deformans Diseases 0.000 description 1
- 102000004067 Osteocalcin Human genes 0.000 description 1
- 108090000573 Osteocalcin Proteins 0.000 description 1
- 206010049088 Osteopenia Diseases 0.000 description 1
- 208000027868 Paget disease Diseases 0.000 description 1
- 208000002193 Pain Diseases 0.000 description 1
- 206010033645 Pancreatitis Diseases 0.000 description 1
- 206010033799 Paralysis Diseases 0.000 description 1
- 208000018737 Parkinson disease Diseases 0.000 description 1
- 206010035664 Pneumonia Diseases 0.000 description 1
- 206010036105 Polyneuropathy Diseases 0.000 description 1
- 208000001280 Prediabetic State Diseases 0.000 description 1
- 238000001069 Raman spectroscopy Methods 0.000 description 1
- 108091027981 Response element Proteins 0.000 description 1
- 208000017442 Retinal disease Diseases 0.000 description 1
- 208000025747 Rheumatic disease Diseases 0.000 description 1
- 206010040047 Sepsis Diseases 0.000 description 1
- 208000021386 Sjogren Syndrome Diseases 0.000 description 1
- PMZURENOXWZQFD-UHFFFAOYSA-L Sodium Sulfate Chemical compound [Na+].[Na+].[O-]S([O-])(=O)=O PMZURENOXWZQFD-UHFFFAOYSA-L 0.000 description 1
- DWAQJAXMDSEUJJ-UHFFFAOYSA-M Sodium bisulfite Chemical compound [Na+].OS([O-])=O DWAQJAXMDSEUJJ-UHFFFAOYSA-M 0.000 description 1
- 208000013200 Stress disease Diseases 0.000 description 1
- 208000006011 Stroke Diseases 0.000 description 1
- 208000030886 Traumatic Brain injury Diseases 0.000 description 1
- 206010069363 Traumatic lung injury Diseases 0.000 description 1
- 206010067584 Type 1 diabetes mellitus Diseases 0.000 description 1
- 230000005856 abnormality Effects 0.000 description 1
- 238000009825 accumulation Methods 0.000 description 1
- 239000002253 acid Substances 0.000 description 1
- 230000009471 action Effects 0.000 description 1
- 230000004913 activation Effects 0.000 description 1
- 210000000577 adipose tissue Anatomy 0.000 description 1
- 230000032683 aging Effects 0.000 description 1
- 239000000556 agonist Substances 0.000 description 1
- 229940083712 aldosterone antagonist Drugs 0.000 description 1
- 230000007815 allergy Effects 0.000 description 1
- 210000004727 amygdala Anatomy 0.000 description 1
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 description 1
- 239000002260 anti-inflammatory agent Substances 0.000 description 1
- 238000013459 approach Methods 0.000 description 1
- 206010003119 arrhythmia Diseases 0.000 description 1
- 230000006793 arrhythmia Effects 0.000 description 1
- 230000004872 arterial blood pressure Effects 0.000 description 1
- 206010003246 arthritis Diseases 0.000 description 1
- 230000001174 ascending effect Effects 0.000 description 1
- 230000008236 biological pathway Effects 0.000 description 1
- 230000031018 biological processes and functions Effects 0.000 description 1
- 230000005540 biological transmission Effects 0.000 description 1
- 230000004097 bone metabolism Effects 0.000 description 1
- 201000011510 cancer Diseases 0.000 description 1
- 125000002915 carbonyl group Chemical group [*:2]C([*:1])=O 0.000 description 1
- 230000015556 catabolic process Effects 0.000 description 1
- 230000005779 cell damage Effects 0.000 description 1
- 238000000701 chemical imaging Methods 0.000 description 1
- 238000009225 cognitive behavioral therapy Methods 0.000 description 1
- 206010009887 colitis Diseases 0.000 description 1
- 201000006815 congenital muscular dystrophy Diseases 0.000 description 1
- 208000027744 congestion Diseases 0.000 description 1
- 208000006111 contracture Diseases 0.000 description 1
- 238000001816 cooling Methods 0.000 description 1
- NWFNSTOSIVLCJA-UHFFFAOYSA-L copper;diacetate;hydrate Chemical compound O.[Cu+2].CC([O-])=O.CC([O-])=O NWFNSTOSIVLCJA-UHFFFAOYSA-L 0.000 description 1
- 238000013480 data collection Methods 0.000 description 1
- 231100000895 deafness Toxicity 0.000 description 1
- 230000002950 deficient Effects 0.000 description 1
- 239000007857 degradation product Substances 0.000 description 1
- 238000013461 design Methods 0.000 description 1
- 238000005828 desilylation reaction Methods 0.000 description 1
- 238000004807 desolvation Methods 0.000 description 1
- 238000000113 differential scanning calorimetry Methods 0.000 description 1
- 238000009792 diffusion process Methods 0.000 description 1
- 208000028659 discharge Diseases 0.000 description 1
- 230000005750 disease progression Effects 0.000 description 1
- 201000009338 distal myopathy Diseases 0.000 description 1
- 238000009826 distribution Methods 0.000 description 1
- 239000002552 dosage form Substances 0.000 description 1
- 231100000371 dose-limiting toxicity Toxicity 0.000 description 1
- 238000001647 drug administration Methods 0.000 description 1
- 238000001035 drying Methods 0.000 description 1
- 208000002169 ectodermal dysplasia Diseases 0.000 description 1
- 208000031068 ectodermal dysplasia syndrome Diseases 0.000 description 1
- 238000002003 electron diffraction Methods 0.000 description 1
- 239000000839 emulsion Substances 0.000 description 1
- 229960001208 eplerenone Drugs 0.000 description 1
- JUKPWJGBANNWMW-VWBFHTRKSA-N eplerenone Chemical compound C([C@@H]1[C@]2(C)C[C@H]3O[C@]33[C@@]4(C)CCC(=O)C=C4C[C@H]([C@@H]13)C(=O)OC)C[C@@]21CCC(=O)O1 JUKPWJGBANNWMW-VWBFHTRKSA-N 0.000 description 1
- 235000019439 ethyl acetate Nutrition 0.000 description 1
- 238000001704 evaporation Methods 0.000 description 1
- 230000008020 evaporation Effects 0.000 description 1
- 230000007717 exclusion Effects 0.000 description 1
- 230000001747 exhibiting effect Effects 0.000 description 1
- 210000003414 extremity Anatomy 0.000 description 1
- 230000001815 facial effect Effects 0.000 description 1
- 206010016256 fatigue Diseases 0.000 description 1
- 230000002349 favourable effect Effects 0.000 description 1
- 230000001605 fetal effect Effects 0.000 description 1
- 208000030941 fetal growth restriction Diseases 0.000 description 1
- 238000001914 filtration Methods 0.000 description 1
- 239000000796 flavoring agent Substances 0.000 description 1
- 235000013355 food flavoring agent Nutrition 0.000 description 1
- 238000009472 formulation Methods 0.000 description 1
- 238000011990 functional testing Methods 0.000 description 1
- 230000004927 fusion Effects 0.000 description 1
- 230000005021 gait Effects 0.000 description 1
- 230000002496 gastric effect Effects 0.000 description 1
- 231100000852 glomerular disease Toxicity 0.000 description 1
- 229950000084 golodirsen Drugs 0.000 description 1
- 208000037824 growth disorder Diseases 0.000 description 1
- 231100000869 headache Toxicity 0.000 description 1
- 208000016354 hearing loss disease Diseases 0.000 description 1
- 208000019622 heart disease Diseases 0.000 description 1
- 208000025070 hereditary periodic fever syndrome Diseases 0.000 description 1
- 229960000890 hydrocortisone Drugs 0.000 description 1
- 125000002887 hydroxy group Chemical group [H]O* 0.000 description 1
- 230000037315 hyperhidrosis Effects 0.000 description 1
- 230000001506 immunosuppresive effect Effects 0.000 description 1
- 238000000338 in vitro Methods 0.000 description 1
- 238000001727 in vivo Methods 0.000 description 1
- 208000027866 inflammatory disease Diseases 0.000 description 1
- 238000011221 initial treatment Methods 0.000 description 1
- 208000028774 intestinal disease Diseases 0.000 description 1
- 230000007794 irritation Effects 0.000 description 1
- 208000028867 ischemia Diseases 0.000 description 1
- 230000009191 jumping Effects 0.000 description 1
- 230000003907 kidney function Effects 0.000 description 1
- 201000003723 learning disability Diseases 0.000 description 1
- 239000006194 liquid suspension Substances 0.000 description 1
- 230000003908 liver function Effects 0.000 description 1
- 238000010234 longitudinal analysis Methods 0.000 description 1
- 231100000515 lung injury Toxicity 0.000 description 1
- 206010025135 lupus erythematosus Diseases 0.000 description 1
- 230000014759 maintenance of location Effects 0.000 description 1
- 208000027202 mammary Paget disease Diseases 0.000 description 1
- 235000012054 meals Nutrition 0.000 description 1
- 230000010534 mechanism of action Effects 0.000 description 1
- 238000002483 medication Methods 0.000 description 1
- 238000000048 melt cooling Methods 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
- 230000000936 membranestabilizing effect Effects 0.000 description 1
- 230000002503 metabolic effect Effects 0.000 description 1
- 235000020938 metabolic status Nutrition 0.000 description 1
- 230000037230 mobility Effects 0.000 description 1
- 201000006417 multiple sclerosis Diseases 0.000 description 1
- 238000001964 muscle biopsy Methods 0.000 description 1
- 230000012042 muscle hypertrophy Effects 0.000 description 1
- 208000013465 muscle pain Diseases 0.000 description 1
- 230000035772 mutation Effects 0.000 description 1
- 230000003274 myotonic effect Effects 0.000 description 1
- 210000001087 myotubule Anatomy 0.000 description 1
- 230000002981 neuropathic effect Effects 0.000 description 1
- 201000001119 neuropathy Diseases 0.000 description 1
- 230000007823 neuropathy Effects 0.000 description 1
- 231100000957 no side effect Toxicity 0.000 description 1
- 235000020824 obesity Nutrition 0.000 description 1
- 238000000399 optical microscopy Methods 0.000 description 1
- 210000000056 organ Anatomy 0.000 description 1
- 230000000399 orthopedic effect Effects 0.000 description 1
- 230000003647 oxidation Effects 0.000 description 1
- 238000007254 oxidation reaction Methods 0.000 description 1
- 235000011837 pasties Nutrition 0.000 description 1
- 244000052769 pathogen Species 0.000 description 1
- 230000001717 pathogenic effect Effects 0.000 description 1
- 230000001575 pathological effect Effects 0.000 description 1
- 230000007170 pathology Effects 0.000 description 1
- 210000004197 pelvis Anatomy 0.000 description 1
- 201000001245 periodontitis Diseases 0.000 description 1
- 208000033808 peripheral neuropathy Diseases 0.000 description 1
- 239000008194 pharmaceutical composition Substances 0.000 description 1
- 230000000144 pharmacologic effect Effects 0.000 description 1
- 238000011458 pharmacological treatment Methods 0.000 description 1
- 230000036470 plasma concentration Effects 0.000 description 1
- 230000007824 polyneuropathy Effects 0.000 description 1
- 230000023603 positive regulation of transcription initiation, DNA-dependent Effects 0.000 description 1
- 239000000843 powder Substances 0.000 description 1
- 201000009104 prediabetes syndrome Diseases 0.000 description 1
- 230000008569 process Effects 0.000 description 1
- 239000000651 prodrug Substances 0.000 description 1
- 229940002612 prodrug Drugs 0.000 description 1
- 230000000770 proinflammatory effect Effects 0.000 description 1
- 230000000069 prophylactic effect Effects 0.000 description 1
- 238000011321 prophylaxis Methods 0.000 description 1
- 230000001681 protective effect Effects 0.000 description 1
- 230000037047 psychomotor activity Effects 0.000 description 1
- 230000010346 psychosocial stress Effects 0.000 description 1
- 208000002815 pulmonary hypertension Diseases 0.000 description 1
- 238000004445 quantitative analysis Methods 0.000 description 1
- 239000011541 reaction mixture Substances 0.000 description 1
- 230000009257 reactivity Effects 0.000 description 1
- 230000010410 reperfusion Effects 0.000 description 1
- 208000023504 respiratory system disease Diseases 0.000 description 1
- 230000000630 rising effect Effects 0.000 description 1
- 238000012502 risk assessment Methods 0.000 description 1
- 229920006395 saturated elastomer Polymers 0.000 description 1
- 206010039722 scoliosis Diseases 0.000 description 1
- 230000035945 sensitivity Effects 0.000 description 1
- 239000000377 silicon dioxide Substances 0.000 description 1
- 238000004467 single crystal X-ray diffraction Methods 0.000 description 1
- 201000009890 sinusitis Diseases 0.000 description 1
- 230000009645 skeletal growth Effects 0.000 description 1
- 208000017520 skin disease Diseases 0.000 description 1
- 201000002859 sleep apnea Diseases 0.000 description 1
- 230000003860 sleep quality Effects 0.000 description 1
- 238000010583 slow cooling Methods 0.000 description 1
- 229910000030 sodium bicarbonate Inorganic materials 0.000 description 1
- 235000017557 sodium bicarbonate Nutrition 0.000 description 1
- 235000010267 sodium hydrogen sulphite Nutrition 0.000 description 1
- 229910052938 sodium sulfate Inorganic materials 0.000 description 1
- 235000011152 sodium sulphate Nutrition 0.000 description 1
- 210000004872 soft tissue Anatomy 0.000 description 1
- 238000000371 solid-state nuclear magnetic resonance spectroscopy Methods 0.000 description 1
- 239000012453 solvate Substances 0.000 description 1
- 208000020431 spinal cord injury Diseases 0.000 description 1
- 229960002256 spironolactone Drugs 0.000 description 1
- LXMSZDCAJNLERA-ZHYRCANASA-N spironolactone Chemical compound C([C@@H]1[C@]2(C)CC[C@@H]3[C@@]4(C)CCC(=O)C=C4C[C@H]([C@@H]13)SC(=O)C)C[C@@]21CCC(=O)O1 LXMSZDCAJNLERA-ZHYRCANASA-N 0.000 description 1
- 208000026841 staggering gait Diseases 0.000 description 1
- 238000010561 standard procedure Methods 0.000 description 1
- 238000011272 standard treatment Methods 0.000 description 1
- 238000013179 statistical model Methods 0.000 description 1
- 238000000528 statistical test Methods 0.000 description 1
- 230000000638 stimulation Effects 0.000 description 1
- 230000036435 stunted growth Effects 0.000 description 1
- 238000000859 sublimation Methods 0.000 description 1
- 230000008022 sublimation Effects 0.000 description 1
- 230000008685 targeting Effects 0.000 description 1
- 230000004797 therapeutic response Effects 0.000 description 1
- 210000003371 toe Anatomy 0.000 description 1
- 230000005758 transcription activity Effects 0.000 description 1
- 230000009466 transformation Effects 0.000 description 1
- 230000007704 transition Effects 0.000 description 1
- 230000009529 traumatic brain injury Effects 0.000 description 1
- 238000011269 treatment regimen Methods 0.000 description 1
- 201000008827 tuberculosis Diseases 0.000 description 1
- 208000001072 type 2 diabetes mellitus Diseases 0.000 description 1
- 210000001364 upper extremity Anatomy 0.000 description 1
- 210000004291 uterus Anatomy 0.000 description 1
- 238000002460 vibrational spectroscopy Methods 0.000 description 1
- 238000001845 vibrational spectrum Methods 0.000 description 1
- 238000005303 weighing Methods 0.000 description 1
- 210000000707 wrist Anatomy 0.000 description 1
Images
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
- A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
- A61K31/57—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone
- A61K31/573—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Veterinary Medicine (AREA)
- Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Life Sciences & Earth Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Neurology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Organic Chemistry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063081073P | 2020-09-21 | 2020-09-21 | |
| US63/081,073 | 2020-09-21 | ||
| US202163195473P | 2021-06-01 | 2021-06-01 | |
| US63/195,473 | 2021-06-01 | ||
| US202163214908P | 2021-06-25 | 2021-06-25 | |
| US63/214,908 | 2021-06-25 | ||
| PCT/US2021/051274 WO2022061287A1 (en) | 2020-09-21 | 2021-09-21 | TREATMENT OF NF-κB-MEDIATED DISEASE |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| KR20230092905A true KR20230092905A (ko) | 2023-06-26 |
Family
ID=80776432
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020237013258A Pending KR20230092905A (ko) | 2020-09-21 | 2021-09-21 | NF-κB-매개 질환의 치료 |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US20220184098A1 (https=) |
| EP (1) | EP4213855A4 (https=) |
| JP (1) | JP2023543439A (https=) |
| KR (1) | KR20230092905A (https=) |
| AU (1) | AU2021343534A1 (https=) |
| CA (1) | CA3170698A1 (https=) |
| IL (1) | IL301500A (https=) |
| MX (1) | MX2023003258A (https=) |
| WO (1) | WO2022061287A1 (https=) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP4436583A4 (en) * | 2021-11-22 | 2025-04-02 | ReveraGen BioPharma, Inc. | TREATMENT OF NF-kB-MEDIATED DISEASE |
| WO2025199347A1 (en) * | 2024-03-21 | 2025-09-25 | Reveragen Biopharma, Inc. | Treatment of glomerulonephritis |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2006279325B2 (en) * | 2005-08-18 | 2013-06-27 | Accelalox, Inc. | Methods for bone treatment by modulating an arachidonic acid metabolic or signaling pathway |
| CA2855756A1 (en) * | 2011-11-29 | 2013-06-06 | Reveragen Biopharma, Inc. | Non-hormonal steroid modulators of nf-kb for treatment of disease |
| WO2014205045A1 (en) * | 2013-06-18 | 2014-12-24 | Downes, Michael | Methods of treating muscular dystrophy |
| PL4023222T3 (pl) * | 2015-09-08 | 2025-03-24 | The Children's Hospital Of Philadelphia | Nieselektywny aktywator metabotropowych receptorów glutaminianu do leczenia zespołu 22q |
| US11382922B2 (en) * | 2019-03-07 | 2022-07-12 | Reveragen Biopharma, Inc. | Aqueous oral pharmaceutical suspension compositions |
-
2021
- 2021-09-21 WO PCT/US2021/051274 patent/WO2022061287A1/en not_active Ceased
- 2021-09-21 JP JP2023518416A patent/JP2023543439A/ja active Pending
- 2021-09-21 MX MX2023003258A patent/MX2023003258A/es unknown
- 2021-09-21 KR KR1020237013258A patent/KR20230092905A/ko active Pending
- 2021-09-21 IL IL301500A patent/IL301500A/en unknown
- 2021-09-21 AU AU2021343534A patent/AU2021343534A1/en active Pending
- 2021-09-21 EP EP21870412.0A patent/EP4213855A4/en active Pending
- 2021-09-21 CA CA3170698A patent/CA3170698A1/en active Pending
-
2022
- 2022-02-21 US US17/651,879 patent/US20220184098A1/en not_active Abandoned
-
2023
- 2023-08-14 US US18/449,312 patent/US20240139210A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| WO2022061287A1 (en) | 2022-03-24 |
| AU2021343534A1 (en) | 2023-05-11 |
| JP2023543439A (ja) | 2023-10-16 |
| US20220184098A1 (en) | 2022-06-16 |
| CA3170698A1 (en) | 2022-03-24 |
| MX2023003258A (es) | 2023-05-08 |
| IL301500A (en) | 2023-05-01 |
| EP4213855A4 (en) | 2024-10-23 |
| EP4213855A1 (en) | 2023-07-26 |
| US20240139210A1 (en) | 2024-05-02 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US20250082651A1 (en) | Ganaxolone for use in treating genetic epileptic disorders | |
| JP7819094B2 (ja) | ヒトアミノステロールｅｎｔ－０３化合物、それを含む関連組成物、およびそれを使用する方法 | |
| US20240139210A1 (en) | Treatment of nf-kb-mediated disease | |
| EP3206682B1 (en) | Combinations comprising siponimod and laquinimod for the treatment of multiple sclerosis | |
| US20240245711A1 (en) | A 19-nor c3,3-disubstituted c21-n-pyrazolyl steroid for the treatment of major depressive disorder | |
| EP3145513B1 (en) | 3-(4-((4-(morpholinomethyl-benzyl)oxy)-1-oxoisoindolin-2-yl)piperidine-2,6-dione for the treatment of systemic lupus erythematosus | |
| KR20150119227A (ko) | 라퀴니모드에 의한 다발성 경화증의 진행형 형태의 치료 | |
| KR20200061310A (ko) | 히스톤 탈아세틸화효소 6 억제제를 포함하는 약학적 조성물 | |
| US20240299416A1 (en) | TREATMENT OF NF-kB-MEDIATED DISEASE | |
| JP2021522344A5 (https=) | ||
| JP2025185105A (ja) | タンパク質キナーゼ阻害剤としてのヘテロ環化合物の新規塩およびこれらの用途 | |
| AU2015231076B2 (en) | Carboxy-cyclopropyl undecanol compounds for treatment of liver disease and other medical disorders | |
| US20240226117A1 (en) | Treatment of congenital adrenal hypoplasia | |
| CN116507338A (zh) | NF-κB介导的疾病的治疗 | |
| JP2024527992A (ja) | 神経刺激性ステロイドの結晶形 | |
| WO2021188148A1 (en) | Crystalline forms of squalamine | |
| CN107249592A (zh) | 用于治疗阿尔茨海默病的方法 | |
| CN118146176B (zh) | G蛋白偶联受体gpr133/adgrd1激动剂、制备方法及其应用 | |
| US20200038414A1 (en) | Methods of treating constipation using aminosterol compositions | |
| AU2024206778A1 (en) | Methods and compositions for treating hsd-1-mediated disorders | |
| EA052165B1 (ru) | Ганаксолон для использования в лечении наследственных эпилептических заболеваний | |
| EP4430056A1 (en) | 3.beta.-hydroxy, 3.alpha.-ethyl steroids for modulation of the alpha-3 subtype of the gaba-a receptor | |
| Krysiak et al. | Coexistence of Glucocorticoid Resistance and Pseudohypoparathyroidism. |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| PA0105 | International application |
Patent event date: 20230419 Patent event code: PA01051R01D Comment text: International Patent Application |
|
| PG1501 | Laying open of application | ||
| A201 | Request for examination | ||
| PA0201 | Request for examination |
Patent event code: PA02012R01D Patent event date: 20240919 Comment text: Request for Examination of Application |