JP2015502964A5 - - Google Patents
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- JP2015502964A5 JP2015502964A5 JP2014547767A JP2014547767A JP2015502964A5 JP 2015502964 A5 JP2015502964 A5 JP 2015502964A5 JP 2014547767 A JP2014547767 A JP 2014547767A JP 2014547767 A JP2014547767 A JP 2014547767A JP 2015502964 A5 JP2015502964 A5 JP 2015502964A5
- Authority
- JP
- Japan
- Prior art keywords
- component
- lysosomal
- combination according
- disease
- lysosomal storage
- 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.)
- Granted
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- 208000015439 Lysosomal storage disease Diseases 0.000 claims 24
- 108010045758 lysosomal proteins Proteins 0.000 claims 16
- 230000007547 defect Effects 0.000 claims 12
- 230000000694 effects Effects 0.000 claims 10
- 230000015556 catabolic process Effects 0.000 claims 8
- 238000006731 degradation reaction Methods 0.000 claims 8
- JBDGDEWWOUBZPM-XYPYZODXSA-N ambroxol Chemical group NC1=C(Br)C=C(Br)C=C1CN[C@@H]1CC[C@@H](O)CC1 JBDGDEWWOUBZPM-XYPYZODXSA-N 0.000 claims 7
- 150000001875 compounds Chemical class 0.000 claims 6
- 201000010099 disease Diseases 0.000 claims 6
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 6
- 239000002552 dosage form Substances 0.000 claims 6
- LXBIFEVIBLOUGU-UHFFFAOYSA-N 1-deoxynojirimycin Chemical group OCC1NCC(O)C(O)C1O LXBIFEVIBLOUGU-UHFFFAOYSA-N 0.000 claims 5
- LXBIFEVIBLOUGU-JGWLITMVSA-N duvoglustat Chemical compound OC[C@H]1NC[C@H](O)[C@@H](O)[C@@H]1O LXBIFEVIBLOUGU-JGWLITMVSA-N 0.000 claims 5
- 238000000034 method Methods 0.000 claims 5
- 239000000825 pharmaceutical preparation Substances 0.000 claims 5
- 108010006519 Molecular Chaperones Proteins 0.000 claims 4
- 230000002132 lysosomal effect Effects 0.000 claims 4
- JDVVGAQPNNXQDW-WCMLQCRESA-N Castanospermine Natural products O[C@H]1[C@@H](O)[C@H]2[C@@H](O)CCN2C[C@H]1O JDVVGAQPNNXQDW-WCMLQCRESA-N 0.000 claims 3
- JDVVGAQPNNXQDW-TVNFTVLESA-N Castinospermine Chemical compound C1[C@H](O)[C@@H](O)[C@H](O)[C@H]2[C@@H](O)CCN21 JDVVGAQPNNXQDW-TVNFTVLESA-N 0.000 claims 3
- 102000004190 Enzymes Human genes 0.000 claims 3
- 108090000790 Enzymes Proteins 0.000 claims 3
- 201000011442 Metachromatic leukodystrophy Diseases 0.000 claims 3
- 239000002253 acid Substances 0.000 claims 3
- 229960005174 ambroxol Drugs 0.000 claims 3
- 125000001841 imino group Chemical group [H]N=* 0.000 claims 3
- 150000003839 salts Chemical class 0.000 claims 3
- 230000032258 transport Effects 0.000 claims 3
- WQZGKKKJIJFFOK-SVZMEOIVSA-N (+)-Galactose Chemical compound OC[C@H]1OC(O)[C@H](O)[C@@H](O)[C@H]1O WQZGKKKJIJFFOK-SVZMEOIVSA-N 0.000 claims 2
- DRHXTSWSUAJOJZ-FMDGEEDCSA-N (3ar,5r,6s,7r,7ar)-5-(hydroxymethyl)-2-methyl-5,6,7,7a-tetrahydro-3ah-pyrano[3,2-d][1,3]thiazole-6,7-diol Chemical compound S1C(C)=N[C@H]2[C@@H]1O[C@H](CO)[C@@H](O)[C@@H]2O DRHXTSWSUAJOJZ-FMDGEEDCSA-N 0.000 claims 2
- YRBKDBZXOAEMOT-UHFFFAOYSA-N 6-deoxy-DMDP Natural products CC1NC(CO)C(O)C1O YRBKDBZXOAEMOT-UHFFFAOYSA-N 0.000 claims 2
- 102100026189 Beta-galactosidase Human genes 0.000 claims 2
- 102100032487 Beta-mannosidase Human genes 0.000 claims 2
- 201000008892 GM1 Gangliosidosis Diseases 0.000 claims 2
- 102100028496 Galactocerebrosidase Human genes 0.000 claims 2
- 108010042681 Galactosylceramidase Proteins 0.000 claims 2
- 102000004547 Glucosylceramidase Human genes 0.000 claims 2
- 108010017544 Glucosylceramidase Proteins 0.000 claims 2
- 102000053187 Glucuronidase Human genes 0.000 claims 2
- 108010060309 Glucuronidase Proteins 0.000 claims 2
- 229920002527 Glycogen Polymers 0.000 claims 2
- 102000003886 Glycoproteins Human genes 0.000 claims 2
- 108090000288 Glycoproteins Proteins 0.000 claims 2
- 229920002683 Glycosaminoglycan Polymers 0.000 claims 2
- 102000004157 Hydrolases Human genes 0.000 claims 2
- 108090000604 Hydrolases Proteins 0.000 claims 2
- 102100033448 Lysosomal alpha-glucosidase Human genes 0.000 claims 2
- 108010027520 N-Acetylgalactosamine-4-Sulfatase Proteins 0.000 claims 2
- MBLBDJOUHNCFQT-UHFFFAOYSA-N N-acetyl-D-galactosamine Natural products CC(=O)NC(C=O)C(O)C(O)C(O)CO MBLBDJOUHNCFQT-UHFFFAOYSA-N 0.000 claims 2
- FTSCEGKYKXESFF-LXTVHRRPSA-N N-nonyldeoxynojirimycin Chemical compound CCCCCCCCCN1C[C@H](O)[C@@H](O)[C@H](O)[C@H]1CO FTSCEGKYKXESFF-LXTVHRRPSA-N 0.000 claims 2
- 102000005348 Neuraminidase Human genes 0.000 claims 2
- 108010006232 Neuraminidase Proteins 0.000 claims 2
- QPYJXFZUIJOGNX-HSUXUTPPSA-N afegostat Chemical compound OC[C@H]1CNC[C@@H](O)[C@@H]1O QPYJXFZUIJOGNX-HSUXUTPPSA-N 0.000 claims 2
- 102000012086 alpha-L-Fucosidase Human genes 0.000 claims 2
- 108010061314 alpha-L-Fucosidase Proteins 0.000 claims 2
- 108010012864 alpha-Mannosidase Proteins 0.000 claims 2
- 102000019199 alpha-Mannosidase Human genes 0.000 claims 2
- 108010005774 beta-Galactosidase Proteins 0.000 claims 2
- 108010055059 beta-Mannosidase Proteins 0.000 claims 2
- 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 claims 2
- 230000002255 enzymatic effect Effects 0.000 claims 2
- 201000008049 fucosidosis Diseases 0.000 claims 2
- 150000004676 glycans Chemical group 0.000 claims 2
- 229940096919 glycogen Drugs 0.000 claims 2
- 238000004519 manufacturing process Methods 0.000 claims 2
- 230000002503 metabolic effect Effects 0.000 claims 2
- 230000035772 mutation Effects 0.000 claims 2
- IHKWXDCSAKJQKM-SRQGCSHVSA-N n-[(1s,6s,7r,8r,8ar)-1,7,8-trihydroxy-1,2,3,5,6,7,8,8a-octahydroindolizin-6-yl]acetamide Chemical compound O[C@H]1[C@H](O)[C@@H](NC(=O)C)CN2CC[C@H](O)[C@@H]21 IHKWXDCSAKJQKM-SRQGCSHVSA-N 0.000 claims 2
- GBRAQQUMMCVTAV-LXGUWJNJSA-N n-[(3s,4r,5r,6r)-4,5-dihydroxy-6-(hydroxymethyl)piperidin-3-yl]acetamide Chemical compound CC(=O)N[C@H]1CN[C@H](CO)[C@@H](O)[C@@H]1O GBRAQQUMMCVTAV-LXGUWJNJSA-N 0.000 claims 2
- 230000000144 pharmacologic effect Effects 0.000 claims 2
- 150000003408 sphingolipids Chemical class 0.000 claims 2
- 102000035160 transmembrane proteins Human genes 0.000 claims 2
- 108091005703 transmembrane proteins Proteins 0.000 claims 2
- AXNKITZDVRGIRT-XDQVBPFNSA-N (1R,2S,3S,4S)-1,4-bis(hydroxymethyl)-6-octyl-5-oxa-6-azabicyclo[2.1.1]hexane-2,3-diol Chemical compound C(CCCCCCC)N1[C@]2(CO)[C@@H](O)[C@H](O)[C@]1(O2)CO AXNKITZDVRGIRT-XDQVBPFNSA-N 0.000 claims 1
- UPZUHYMBTUUPML-XGUBFFRZSA-N (1S,2S,3R,6S)-4-(hydroxymethyl)-6-(octylamino)cyclohex-4-ene-1,2,3-triol Chemical compound CCCCCCCCN[C@H]1C=C(CO)[C@@H](O)[C@H](O)[C@H]1O UPZUHYMBTUUPML-XGUBFFRZSA-N 0.000 claims 1
- ZEWFPWKROPWRKE-CQOGJGKDSA-N (2r,3r,4r,5r,6s)-2-(hydroxymethyl)-6-methylpiperidine-3,4,5-triol Chemical compound C[C@@H]1N[C@H](CO)[C@@H](O)[C@H](O)[C@@H]1O ZEWFPWKROPWRKE-CQOGJGKDSA-N 0.000 claims 1
- YRBKDBZXOAEMOT-DPYQTVNSSA-N (2s,3r,4s,5r)-2-(hydroxymethyl)-5-methylpyrrolidine-3,4-diol Chemical compound C[C@H]1N[C@@H](CO)[C@@H](O)[C@H]1O YRBKDBZXOAEMOT-DPYQTVNSSA-N 0.000 claims 1
- ZSNFEMNRWFDMNU-CVPUBMOQSA-N (2s,3s,4r,5s,6r)-2-butyl-6-(hydroxymethyl)piperidine-3,4,5-triol Chemical compound CCCC[C@@H]1N[C@H](CO)[C@H](O)[C@H](O)[C@H]1O ZSNFEMNRWFDMNU-CVPUBMOQSA-N 0.000 claims 1
- YRBKDBZXOAEMOT-VANKVMQKSA-N (2s,3s,4s,5r)-2-(hydroxymethyl)-5-methylpyrrolidine-3,4-diol Chemical compound C[C@H]1N[C@@H](CO)[C@H](O)[C@H]1O YRBKDBZXOAEMOT-VANKVMQKSA-N 0.000 claims 1
- QPYJXFZUIJOGNX-PBXRRBTRSA-N (3r,4s,5r)-5-(hydroxymethyl)piperidine-3,4-diol Chemical compound OC[C@H]1CNC[C@@H](O)[C@H]1O QPYJXFZUIJOGNX-PBXRRBTRSA-N 0.000 claims 1
- LXBIFEVIBLOUGU-FSIIMWSLSA-N 1-Deoxymannojirimycin Natural products OC[C@@H]1NC[C@@H](O)[C@H](O)[C@H]1O LXBIFEVIBLOUGU-FSIIMWSLSA-N 0.000 claims 1
- 102000006772 Acid Ceramidase Human genes 0.000 claims 1
- 108020005296 Acid Ceramidase Proteins 0.000 claims 1
- 208000034012 Acid sphingomyelinase deficiency Diseases 0.000 claims 1
- 102100031317 Alpha-N-acetylgalactosaminidase Human genes 0.000 claims 1
- 102100034561 Alpha-N-acetylglucosaminidase Human genes 0.000 claims 1
- 102100022146 Arylsulfatase A Human genes 0.000 claims 1
- 102100031491 Arylsulfatase B Human genes 0.000 claims 1
- 108010023546 Aspartylglucosylaminase Proteins 0.000 claims 1
- 101710124976 Beta-hexosaminidase A Proteins 0.000 claims 1
- 101710124978 Beta-hexosaminidase B Proteins 0.000 claims 1
- 102100022548 Beta-hexosaminidase subunit alpha Human genes 0.000 claims 1
- 108010036867 Cerebroside-Sulfatase Proteins 0.000 claims 1
- 208000024720 Fabry Disease Diseases 0.000 claims 1
- 208000001948 Farber Lipogranulomatosis Diseases 0.000 claims 1
- 208000033149 Farber disease Diseases 0.000 claims 1
- 208000001905 GM2 Gangliosidoses Diseases 0.000 claims 1
- 201000008905 GM2 gangliosidosis Diseases 0.000 claims 1
- IAJILQKETJEXLJ-UHFFFAOYSA-N Galacturonsaeure Natural products O=CC(O)C(O)C(O)C(O)C(O)=O IAJILQKETJEXLJ-UHFFFAOYSA-N 0.000 claims 1
- 208000020322 Gaucher disease type I Diseases 0.000 claims 1
- 208000020916 Gaucher disease type II Diseases 0.000 claims 1
- 208000028735 Gaucher disease type III Diseases 0.000 claims 1
- 208000010055 Globoid Cell Leukodystrophy Diseases 0.000 claims 1
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 claims 1
- 206010053185 Glycogen storage disease type II Diseases 0.000 claims 1
- 229920002971 Heparan sulfate Polymers 0.000 claims 1
- 101001045440 Homo sapiens Beta-hexosaminidase subunit alpha Proteins 0.000 claims 1
- 101001124388 Homo sapiens NPC intracellular cholesterol transporter 1 Proteins 0.000 claims 1
- 102000004627 Iduronidase Human genes 0.000 claims 1
- 108010003381 Iduronidase Proteins 0.000 claims 1
- 208000028226 Krabbe disease Diseases 0.000 claims 1
- 102000004882 Lipase Human genes 0.000 claims 1
- 108090001060 Lipase Proteins 0.000 claims 1
- 108010009491 Lysosomal-Associated Membrane Protein 2 Proteins 0.000 claims 1
- 102000009565 Lysosomal-Associated Membrane Protein 2 Human genes 0.000 claims 1
- 208000027933 Mannosidase Deficiency disease Diseases 0.000 claims 1
- BLOFGONIVNXZME-UHFFFAOYSA-N Mannostatin A Natural products CSC1C(N)C(O)C(O)C1O BLOFGONIVNXZME-UHFFFAOYSA-N 0.000 claims 1
- 208000008955 Mucolipidoses Diseases 0.000 claims 1
- 206010072927 Mucolipidosis type I Diseases 0.000 claims 1
- 102100021003 N(4)-(beta-N-acetylglucosaminyl)-L-asparaginase Human genes 0.000 claims 1
- OVRNDRQMDRJTHS-CBQIKETKSA-N N-Acetyl-D-Galactosamine Chemical compound CC(=O)N[C@H]1[C@@H](O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-CBQIKETKSA-N 0.000 claims 1
- 102000018740 N-Acetylgalactosamine-4-Sulfatase Human genes 0.000 claims 1
- OVRNDRQMDRJTHS-KEWYIRBNSA-N N-acetyl-D-galactosamine Chemical compound CC(=O)N[C@H]1C(O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-KEWYIRBNSA-N 0.000 claims 1
- 108010023320 N-acetylglucosamine-6-sulfatase Proteins 0.000 claims 1
- 108010006140 N-sulfoglucosamine sulfohydrolase Proteins 0.000 claims 1
- 102100027661 N-sulphoglucosamine sulphohydrolase Human genes 0.000 claims 1
- 201000000785 Niemann-Pick disease type C2 Diseases 0.000 claims 1
- BGMYHTUCJVZIRP-UHFFFAOYSA-N Nojirimycin Natural products OCC1NC(O)C(O)C(O)C1O BGMYHTUCJVZIRP-UHFFFAOYSA-N 0.000 claims 1
- 208000018737 Parkinson disease Diseases 0.000 claims 1
- 108091000080 Phosphotransferase Proteins 0.000 claims 1
- 108091006161 SLC17A5 Proteins 0.000 claims 1
- 208000017460 Sialidosis type 2 Diseases 0.000 claims 1
- 102000011971 Sphingomyelin Phosphodiesterase Human genes 0.000 claims 1
- 108010061312 Sphingomyelin Phosphodiesterase Proteins 0.000 claims 1
- 102000005262 Sulfatase Human genes 0.000 claims 1
- QAOWNCQODCNURD-UHFFFAOYSA-L Sulfate Chemical compound [O-]S([O-])(=O)=O QAOWNCQODCNURD-UHFFFAOYSA-L 0.000 claims 1
- 108010044965 UDP-N-acetylglucosamine-lysosomal-enzyme N-acetylglucosaminephosphotransferase Proteins 0.000 claims 1
- 108010070626 acid beta-galactosidase Proteins 0.000 claims 1
- 239000013543 active substance Substances 0.000 claims 1
- DCVWZPYTNWZZIM-UHFFFAOYSA-N alpha-7-deoxyhomonojirimycin Natural products CC1NC(CO)C(O)CC1O DCVWZPYTNWZZIM-UHFFFAOYSA-N 0.000 claims 1
- 108010030291 alpha-Galactosidase Proteins 0.000 claims 1
- 102000005840 alpha-Galactosidase Human genes 0.000 claims 1
- 108010028144 alpha-Glucosidases Proteins 0.000 claims 1
- 108010015684 alpha-N-Acetylgalactosaminidase Proteins 0.000 claims 1
- 108010009380 alpha-N-acetyl-D-glucosaminidase Proteins 0.000 claims 1
- CLVUFWXGNIFGNC-UHFFFAOYSA-N alpha-homonojirimycin Natural products OCC1NC(CO)C(O)C(O)C1O CLVUFWXGNIFGNC-UHFFFAOYSA-N 0.000 claims 1
- ZSNFEMNRWFDMNU-UHFFFAOYSA-N beta-1-C-butyl-deoxygalactonojirimycin Natural products CCCCC1NC(CO)C(O)C(O)C1O ZSNFEMNRWFDMNU-UHFFFAOYSA-N 0.000 claims 1
- 229960003870 bromhexine Drugs 0.000 claims 1
- OJGDCBLYJGHCIH-UHFFFAOYSA-N bromhexine Chemical compound C1CCCCC1N(C)CC1=CC(Br)=CC(Br)=C1N OJGDCBLYJGHCIH-UHFFFAOYSA-N 0.000 claims 1
- CLVUFWXGNIFGNC-OVHBTUCOSA-N chembl501355 Chemical compound OC[C@H]1N[C@H](CO)[C@H](O)[C@@H](O)[C@@H]1O CLVUFWXGNIFGNC-OVHBTUCOSA-N 0.000 claims 1
- 235000012000 cholesterol Nutrition 0.000 claims 1
- 150000001840 cholesterol esters Chemical class 0.000 claims 1
- 230000007812 deficiency Effects 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 229930182830 galactose Natural products 0.000 claims 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 claims 1
- 108010054451 glucosamine acetyltransferase Proteins 0.000 claims 1
- 201000004502 glycogen storage disease II Diseases 0.000 claims 1
- 201000008977 glycoproteinosis Diseases 0.000 claims 1
- IAJILQKETJEXLJ-LECHCGJUSA-N iduronic acid Chemical compound O=C[C@@H](O)[C@H](O)[C@@H](O)[C@H](O)C(O)=O IAJILQKETJEXLJ-LECHCGJUSA-N 0.000 claims 1
- 150000002632 lipids Chemical class 0.000 claims 1
- BLOFGONIVNXZME-YDMGZANHSA-N mannostatin A Chemical compound CS[C@@H]1[C@@H](N)[C@@H](O)[C@@H](O)[C@H]1O BLOFGONIVNXZME-YDMGZANHSA-N 0.000 claims 1
- UQRORFVVSGFNRO-UTINFBMNSA-N miglustat Chemical compound CCCCN1C[C@H](O)[C@@H](O)[C@H](O)[C@H]1CO UQRORFVVSGFNRO-UTINFBMNSA-N 0.000 claims 1
- 208000022018 mucopolysaccharidosis type 2 Diseases 0.000 claims 1
- 208000011045 mucopolysaccharidosis type 3 Diseases 0.000 claims 1
- BGMYHTUCJVZIRP-GASJEMHNSA-N nojirimycin Chemical compound OC[C@H]1NC(O)[C@H](O)[C@@H](O)[C@@H]1O BGMYHTUCJVZIRP-GASJEMHNSA-N 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 102000020233 phosphotransferase Human genes 0.000 claims 1
- 229920001184 polypeptide Polymers 0.000 claims 1
- 230000002265 prevention Effects 0.000 claims 1
- 102000004196 processed proteins & peptides Human genes 0.000 claims 1
- 108090000765 processed proteins & peptides Proteins 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- WKSAUQYGYAYLPV-UHFFFAOYSA-N pyrimethamine Chemical compound CCC1=NC(N)=NC(N)=C1C1=CC=C(Cl)C=C1 WKSAUQYGYAYLPV-UHFFFAOYSA-N 0.000 claims 1
- KNSGUARWFVOEEB-UHFFFAOYSA-N pyrrolo[3,4-d]pyridazin-4-one Chemical compound O=C1N=NC=C2C=NC=C12 KNSGUARWFVOEEB-UHFFFAOYSA-N 0.000 claims 1
- 208000011985 sialidosis Diseases 0.000 claims 1
- 239000012453 solvate Substances 0.000 claims 1
- 108060007951 sulfatase Proteins 0.000 claims 1
- FXUAIOOAOAVCGD-UHFFFAOYSA-N swainsonine Natural products C1CCC(O)C2C(O)C(O)CN21 FXUAIOOAOAVCGD-UHFFFAOYSA-N 0.000 claims 1
- FXUAIOOAOAVCGD-FKSUSPILSA-N swainsonine Chemical compound C1CC[C@H](O)[C@H]2[C@H](O)[C@H](O)CN21 FXUAIOOAOAVCGD-FKSUSPILSA-N 0.000 claims 1
- 229960005566 swainsonine Drugs 0.000 claims 1
- 230000001225 therapeutic effect Effects 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP11010078.1 | 2011-12-22 | ||
| EP11010078 | 2011-12-22 | ||
| PCT/EP2012/005363 WO2013091897A1 (en) | 2011-12-22 | 2012-12-21 | Combination of a compound having the ability to rearrange a lysosomal enzyme and ambroxol and/or a derivative of ambroxol |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017248953A Division JP2018076353A (ja) | 2011-12-22 | 2017-12-26 | リソソーム酵素を再構成する能力を有する化合物ならびにアンブロキソールおよび／またはアンブロキソールの誘導体の組み合わせ |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2015502964A JP2015502964A (ja) | 2015-01-29 |
| JP2015502964A5 true JP2015502964A5 (https=) | 2016-01-28 |
| JP6320931B2 JP6320931B2 (ja) | 2018-05-09 |
Family
ID=47559375
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2014547767A Expired - Fee Related JP6320931B2 (ja) | 2011-12-22 | 2012-12-21 | リソソーム酵素を再構成する能力を有する化合物ならびにアンブロキソールおよび／またはアンブロキソールの誘導体の組み合わせ |
| JP2017248953A Pending JP2018076353A (ja) | 2011-12-22 | 2017-12-26 | リソソーム酵素を再構成する能力を有する化合物ならびにアンブロキソールおよび／またはアンブロキソールの誘導体の組み合わせ |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017248953A Pending JP2018076353A (ja) | 2011-12-22 | 2017-12-26 | リソソーム酵素を再構成する能力を有する化合物ならびにアンブロキソールおよび／またはアンブロキソールの誘導体の組み合わせ |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US20150258081A1 (https=) |
| EP (1) | EP2793872B1 (https=) |
| JP (2) | JP6320931B2 (https=) |
| KR (1) | KR102076585B1 (https=) |
| AU (2) | AU2012359165A1 (https=) |
| BR (1) | BR112014014808A2 (https=) |
| CA (1) | CA2860085A1 (https=) |
| ES (1) | ES2864774T3 (https=) |
| IL (1) | IL233173B (https=) |
| WO (1) | WO2013091897A1 (https=) |
Families Citing this family (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9675627B2 (en) * | 2014-04-14 | 2017-06-13 | Amicus Therapeutics, Inc. | Dosing regimens for treating and/or preventing cerebral amyloidoses |
| CN114540327B (zh) | 2014-09-30 | 2026-03-24 | 阿米库斯治疗学公司 | 具有增强的碳水化合物的高强度酸性α-葡糖苷酶 |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| JP6981961B2 (ja) * | 2015-07-30 | 2021-12-17 | ホライズン オルファン エルエルシー | フコシダーゼ阻害剤 |
| KR20250007693A (ko) | 2015-12-30 | 2025-01-14 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병 치료용의 강화된 산 알파-글루코시다제 |
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| KR20220044560A (ko) | 2019-08-07 | 2022-04-08 | 아미쿠스 세라퓨틱스, 인코포레이티드 | Gla 유전자에 돌연변이를 갖는 환자에서 파브리 질병을 치료하는 방법 |
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| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DE19933148A1 (de) | 1999-07-20 | 2001-01-25 | Boehringer Ingelheim Int | Ambroxolhaltige Lutschtablette |
| EP2857036B1 (en) * | 2003-01-31 | 2016-12-21 | Mount Sinai School of Medicine of New York University | Combination therapy for treating protein deficiency disorders |
| PL2533050T6 (pl) * | 2006-05-16 | 2016-05-31 | Amicus Therapeutics Inc | Możliwości leczenia choroby Fabry'ego |
| JP5337405B2 (ja) * | 2007-09-17 | 2013-11-06 | ザ・ホスピタル・フォー・シック・チルドレン | ゴーシェ病の治療方法 |
| CA2718182A1 (en) * | 2008-03-12 | 2009-09-17 | Amicus Therapeutics, Inc. | Treatment of pompe disease with specific pharmacological chaperones and monitoring treatment using surrogate markers |
| EP2353403B1 (de) * | 2010-02-01 | 2012-07-11 | Symrise AG | Verwendung von 1-(2,4-Dihydroxy-phenyl)-3-(3-hydroxy-4-methoxy-phenyl)-propan-1-on |
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- 2012-12-21 EP EP12815644.5A patent/EP2793872B1/en not_active Not-in-force
- 2012-12-21 AU AU2012359165A patent/AU2012359165A1/en not_active Abandoned
- 2012-12-21 ES ES12815644T patent/ES2864774T3/es active Active
- 2012-12-21 JP JP2014547767A patent/JP6320931B2/ja not_active Expired - Fee Related
- 2012-12-21 WO PCT/EP2012/005363 patent/WO2013091897A1/en not_active Ceased
- 2012-12-21 BR BR112014014808A patent/BR112014014808A2/pt not_active Application Discontinuation
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2014
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2017
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