LU93312I2 - Migalastat ou un sel de celui-ci, y compris le sel de chlorhydrate - Google Patents
Migalastat ou un sel de celui-ci, y compris le sel de chlorhydrate Download PDFInfo
- Publication number
- LU93312I2 LU93312I2 LU93312C LU93312C LU93312I2 LU 93312 I2 LU93312 I2 LU 93312I2 LU 93312 C LU93312 C LU 93312C LU 93312 C LU93312 C LU 93312C LU 93312 I2 LU93312 I2 LU 93312I2
- Authority
- LU
- Luxembourg
- Prior art keywords
- salt
- migalastat
- chlorhydrate
- including salt
- Prior art date
Links
Classifications
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- 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
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
- A61K31/445—Non condensed piperidines, e.g. piperocaine
-
- 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
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
- A61K31/445—Non condensed piperidines, e.g. piperocaine
- A61K31/45—Non condensed piperidines, e.g. piperocaine having oxo groups directly attached to the heterocyclic ring, e.g. cycloheximide
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
-
- 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
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D211/00—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
- C07D211/04—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
- C07D211/06—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members
- C07D211/36—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
- C07D211/40—Oxygen atoms
- C07D211/44—Oxygen atoms attached in position 4
- C07D211/46—Oxygen atoms attached in position 4 having a hydrogen atom as the second substituent in position 4
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
- G01N33/5094—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for blood cell populations
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
- G01N2333/90—Enzymes; Proenzymes
- G01N2333/914—Hydrolases (3)
- G01N2333/924—Hydrolases (3) acting on glycosyl compounds (3.2)
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
- G01N2333/90—Enzymes; Proenzymes
- G01N2333/914—Hydrolases (3)
- G01N2333/924—Hydrolases (3) acting on glycosyl compounds (3.2)
- G01N2333/94—Hydrolases (3) acting on glycosyl compounds (3.2) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2800/00—Detection or diagnosis of diseases
- G01N2800/04—Endocrine or metabolic disorders
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2800/00—Detection or diagnosis of diseases
- G01N2800/52—Predicting or monitoring the response to treatment, e.g. for selection of therapy based on assay results in personalised medicine; Prognosis
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Epidemiology (AREA)
- Immunology (AREA)
- Urology & Nephrology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Hematology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Cell Biology (AREA)
- Food Science & Technology (AREA)
- Biochemistry (AREA)
- Analytical Chemistry (AREA)
- Pathology (AREA)
- Ecology (AREA)
- General Physics & Mathematics (AREA)
- Tropical Medicine & Parasitology (AREA)
- Physics & Mathematics (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Heart & Thoracic Surgery (AREA)
- Cardiology (AREA)
- Physiology (AREA)
- Nutrition Science (AREA)
- Vascular Medicine (AREA)
- Diabetes (AREA)
- Obesity (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US80108906P | 2006-05-16 | 2006-05-16 | |
| US85363106P | 2006-10-23 | 2006-10-23 | |
| PCT/US2007/069046 WO2007137072A2 (fr) | 2006-05-16 | 2007-05-16 | Dosages biologiques pour le diagnostic et l'évaluation d'options de traitement de la maladie de fabry |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| LU93312I2 true LU93312I2 (fr) | 2017-01-26 |
Family
ID=38723987
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| LU93312C LU93312I2 (fr) | 2006-05-16 | 2016-11-16 | Migalastat ou un sel de celui-ci, y compris le sel de chlorhydrate |
Country Status (19)
| Country | Link |
|---|---|
| US (9) | US7851143B2 (fr) |
| EP (6) | EP2533050B3 (fr) |
| JP (7) | JP2009537149A (fr) |
| AU (1) | AU2007253900A1 (fr) |
| CA (1) | CA2652553C (fr) |
| CY (3) | CY1115180T1 (fr) |
| DK (3) | DK2533050T6 (fr) |
| ES (4) | ES2464527T7 (fr) |
| FI (1) | FI3457135T3 (fr) |
| HK (1) | HK1218955A1 (fr) |
| HU (3) | HUE071237T2 (fr) |
| LT (2) | LT3457135T (fr) |
| LU (1) | LU93312I2 (fr) |
| MX (2) | MX351004B (fr) |
| NL (1) | NL300843I2 (fr) |
| PL (3) | PL2787345T3 (fr) |
| PT (2) | PT3457135T (fr) |
| SI (3) | SI3457135T1 (fr) |
| WO (1) | WO2007137072A2 (fr) |
Families Citing this family (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2533050B3 (fr) | 2006-05-16 | 2015-06-24 | Amicus Therapeutics, Inc. | Options de traitement pour la maladie de Fabry |
| MX2009011473A (es) * | 2007-04-26 | 2010-01-18 | Amicus Therapeutics Inc | Regimenes de dosificacion para el tratamiento de enfermedades por almacenamiento lisosomal que utilizan chaperonas farmacologicas. |
| US9999618B2 (en) | 2007-04-26 | 2018-06-19 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
| SI2252313T1 (sl) | 2008-02-12 | 2015-08-31 | Amicus Therapeutics, Inc. | Postopek napovedovanja odgovora na zdravljenje bolezni s farmakološkim spremljevalcem |
| MX2010009875A (es) * | 2008-03-12 | 2010-11-26 | Amicus Therapeutics Inc | Ensayos para diagnosticar y evaluar opciones de tratamiento para enfermedad de pompe. |
| EP2502068B1 (fr) * | 2009-11-17 | 2016-02-10 | Baylor Research Institute | Triaosylceramide urinaire (gb3) comme marqueur de maladie cardiaque |
| AU2011223706A1 (en) * | 2010-03-05 | 2012-09-20 | Baylor Research Institute | Involvement of androgen/androgen receptor pathway in Fabry disease |
| CN107088225A (zh) * | 2011-03-11 | 2017-08-25 | 阿米库斯治疗学公司 | 治疗法布里病的给药方案 |
| WO2012154967A1 (fr) | 2011-05-12 | 2012-11-15 | Proteostasis Therapeutics, Inc. | Régulateurs de la protéostasie |
| BR112014014808A2 (pt) * | 2011-12-22 | 2018-09-25 | Centogene Ip Gmbh | combinações de um composto que tem a capacidade de reorganizar uma enzima lisossômica e ambroxol e/ou um derivado de ambroxol, sua utilização, preparação farmacêutica e seu método de preparação |
| EP2806875B1 (fr) | 2012-01-25 | 2017-07-19 | Proteostasis Therapeutics, Inc. | Composés permettant de moduler l'activité de protéasome |
| WO2014127038A1 (fr) * | 2013-02-12 | 2014-08-21 | Baylor Research Institute | Procédés et compositions associés à l'homéostase du calcium et la maladie de fabry |
| ES2732308T3 (es) * | 2013-12-23 | 2019-11-21 | Bcn Peptides Sa | Análogos de bicalutamida o (s)-bicalutamida como compuestos activadores de la exocitosis para su uso en el tratamiento de un desorden de acúmulo lisosomal o glucogenosis |
| US9675627B2 (en) * | 2014-04-14 | 2017-06-13 | Amicus Therapeutics, Inc. | Dosing regimens for treating and/or preventing cerebral amyloidoses |
| ES2715889T3 (es) | 2014-07-25 | 2019-06-06 | Shire Human Genetic Therapies | Estructura cristalina del dominio de tipo proteína de transferencia de glicolípidos de la proteína adaptadora de cuatro fosfatos 2 humana |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| CA3031249A1 (fr) * | 2016-07-19 | 2018-01-25 | Amicus Therapeutics, Inc. | Traitement de la maladie de fabry chez des patients soumis a une therapie enzymatique substitutive (ert) et chez des patients non soumis a une ert |
| US12020820B1 (en) | 2017-03-03 | 2024-06-25 | Cerner Innovation, Inc. | Predicting sphingolipidoses (fabry's disease) and decision support |
| US11335461B1 (en) | 2017-03-06 | 2022-05-17 | Cerner Innovation, Inc. | Predicting glycogen storage diseases (Pompe disease) and decision support |
| KR102631805B1 (ko) | 2017-05-30 | 2024-01-31 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 신장 손상을 갖는 파브리 환자를 치료하는 방법 |
| EP3630114B1 (fr) * | 2017-05-30 | 2023-11-01 | Amicus Therapeutics, Inc. | Migalastat pour le traitement de patients atteints de la maladie de fabry souffrants d'une insuffisance rénale |
| US11923048B1 (en) | 2017-10-03 | 2024-03-05 | Cerner Innovation, Inc. | Determining mucopolysaccharidoses and decision support tool |
| JP7555818B2 (ja) | 2018-02-06 | 2024-09-25 | アミカス セラピューティックス インコーポレイテッド | 妊娠患者におけるファブリー病の治療のためのミガラスタットの使用 |
| HUE064761T2 (hu) * | 2018-02-06 | 2024-04-28 | Amicus Therapeutics Inc | Klasszikus FABRY-betegségben szenvedõ betegek kezelése migalasztáttal |
| WO2020006269A1 (fr) | 2018-06-27 | 2020-01-02 | Proteostasis Therapeutics, Inc. | Composés améliorant l'activité du protéasome |
| MX2020014082A (es) | 2018-06-27 | 2021-04-13 | Proteostasis Therapeutics Inc | Compuestos que mejoran la actividad del proteasoma. |
| US20220184185A1 (en) | 2018-07-25 | 2022-06-16 | Modernatx, Inc. | Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders |
| HRP20260109T1 (hr) | 2019-08-07 | 2026-03-13 | Amicus Therapeutics, Inc. | Migalastat za upotrebu u liječenju fabryjeve bolesti kod pacijenata koji imaju mutaciju u gla genu |
| US11191827B1 (en) | 2020-12-09 | 2021-12-07 | Tevogen Bio Inc. | COVID-19 peptide specific T-cells and methods of treating and preventing COVID-19 |
| US11623916B2 (en) | 2020-12-16 | 2023-04-11 | Amicus Therapeutics, Inc. | Highly purified batches of pharmaceutical grade migalastat and methods of producing the same |
Family Cites Families (40)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0463109A4 (en) * | 1989-03-24 | 1992-11-19 | Research Corporation Technologies, Inc. | Recombinant alpha-galactosidase, a therapy for fabry disease |
| WO1992000277A1 (fr) * | 1990-06-29 | 1992-01-09 | Nippon Shinyaku Co., Ltd. | Derive de piperidine |
| EP0959877A4 (fr) | 1996-04-10 | 2000-08-23 | Univ California | Correction des defaillances genetiques a l'aide de chaperons chimiques |
| US6270954B1 (en) | 1996-04-10 | 2001-08-07 | The Regents Of The University Of California | Correction of genetic defects using chemical chaperones |
| US6274597B1 (en) | 1998-06-01 | 2001-08-14 | Mount Sinai School Of Medicine Of New York University | Method of enhancing lysosomal α-Galactosidase A |
| US7842470B2 (en) | 2001-10-09 | 2010-11-30 | Oregon Health & Science University | Method for pharmacoperones correction of GnRHR mutant protein misfolding |
| CN102586205A (zh) | 2003-01-31 | 2012-07-18 | 纽约大学西奈山医学院 | 酶和酶的活性部位特异性陪伴分子的组合 |
| US7446098B2 (en) * | 2003-02-18 | 2008-11-04 | Mount Sinai School Of Medicine Of New York University | Combination therapy for treating protein deficiencies |
| US20070066543A1 (en) * | 2003-05-22 | 2007-03-22 | The Hospital For Sick Children | Treatment of tay sachs or sandhoff diseases by enhancing hexosaminidase activity |
| FR2861991B1 (fr) | 2003-11-07 | 2008-01-18 | Centre Nat Rech Scient | Utilisation d'inhibiteurs de glucosidase pour une therapie de la mucoviscidose |
| BRPI0416216A (pt) | 2003-11-12 | 2006-12-26 | Amicus Therapeutics Inc | composto de glicoimidazol e polihidroxiciclohexenila e método para sintetizar composto de glicoimidazol e polihidroxiciclohexenila, métodos para inibir glicocerebrosidase, para aumentar a atividade de glicocerebrosidase e para estabilizar glicocerebrosidade, composição farmacêutica, métodos para inibir uma -glicosidade, e para tratar doença de gaucher, derivado de glicoimidazol e derivados de polihidroxiciclohexilamina |
| EP3782655A1 (fr) | 2005-05-17 | 2021-02-24 | Amicus Therapeutics, Inc. | Procédé pour le traitement de la maladie de pompe au moyen de 1-désoxynojirimycine et de ses dérivés |
| WO2008045015A1 (fr) | 2005-06-08 | 2008-04-17 | Amicus Therapeutics, Inc. | Purification d'imino- et amino-sucre |
| WO2006133446A2 (fr) | 2005-06-08 | 2006-12-14 | Amicus Therapeutics, Inc. | Traitement de troubles du snc associes a des mutations dans des genes codant pour des enzymes lysosomales |
| AU2006272497B2 (en) | 2005-07-27 | 2012-07-19 | University Of Florida Research Foundation, Inc. | Small compounds that correct protein misfolding and uses thereof |
| WO2007089591A2 (fr) | 2006-01-27 | 2007-08-09 | The Board Of Trustees Of The Leland Stanford Junior University | Compositions et méthodes pour criblage à haut débit de chaperons pharmacologiques |
| EP2533050B3 (fr) | 2006-05-16 | 2015-06-24 | Amicus Therapeutics, Inc. | Options de traitement pour la maladie de Fabry |
| DE602007012924D1 (de) | 2006-09-22 | 2011-04-14 | Krka Tovarna Zdravil D D | Aripiprazolhemifumarat und verfahren zu dessen herstellung |
| CA2682441A1 (fr) | 2007-03-30 | 2008-10-09 | Amicus Therapeutics, Inc. | Procede pour le traitement de la maladie de fabry utilisant des chaperons pharmacologiques |
| MX2009011473A (es) | 2007-04-26 | 2010-01-18 | Amicus Therapeutics Inc | Regimenes de dosificacion para el tratamiento de enfermedades por almacenamiento lisosomal que utilizan chaperonas farmacologicas. |
| US9999618B2 (en) | 2007-04-26 | 2018-06-19 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
| SI2252313T1 (sl) | 2008-02-12 | 2015-08-31 | Amicus Therapeutics, Inc. | Postopek napovedovanja odgovora na zdravljenje bolezni s farmakološkim spremljevalcem |
| WO2010048532A1 (fr) | 2008-10-24 | 2010-04-29 | Amicus Therapeutics, Inc. | Modèle de dosage à multiples compartiments |
| US9206457B2 (en) | 2009-05-26 | 2015-12-08 | Amicus Therapeutics, Inc. | Utilization of pharmacological chaperones to improve manufacturing and purification of biologics |
| JP5805630B2 (ja) | 2009-05-26 | 2015-11-04 | アミカス セラピューティックス インコーポレイテッド | 生物製剤の製造および精製を改善するための薬理学的シャペロンの利用 |
| EP2502068B1 (fr) | 2009-11-17 | 2016-02-10 | Baylor Research Institute | Triaosylceramide urinaire (gb3) comme marqueur de maladie cardiaque |
| CN107088225A (zh) | 2011-03-11 | 2017-08-25 | 阿米库斯治疗学公司 | 治疗法布里病的给药方案 |
| US10155027B2 (en) | 2012-07-17 | 2018-12-18 | Amicus Therapeutics, Inc. | Alpha-galactosidase A and 1-deoxygalactonojirimycin co-formulation for the treatment of fabry disease |
| EP2874648A4 (fr) | 2012-07-17 | 2015-12-30 | Amicus Therapeutics Inc | Co-formulation d'alpha-galactosidase a et de 1-désoxygalactonojirimycine |
| JP7098529B2 (ja) | 2016-03-22 | 2022-07-11 | アミカス セラピューティックス インコーポレイテッド | Gla遺伝子にg9331a変異を有する患者においてファブリー病を処置する方法 |
| CA3031249A1 (fr) | 2016-07-19 | 2018-01-25 | Amicus Therapeutics, Inc. | Traitement de la maladie de fabry chez des patients soumis a une therapie enzymatique substitutive (ert) et chez des patients non soumis a une ert |
| US11357834B2 (en) | 2017-01-10 | 2022-06-14 | Amicus Therapeutics, Inc. | Recombinant α-galactosidase A for treatment of Fabry disease |
| KR102631805B1 (ko) | 2017-05-30 | 2024-01-31 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 신장 손상을 갖는 파브리 환자를 치료하는 방법 |
| SG11202000423XA (en) | 2017-07-19 | 2020-02-27 | Amicus Therapeutics Inc | Treatment of fabry disease in ert-naïve and ert-experienced patients |
| SMT202200459T1 (it) | 2017-08-28 | 2023-01-13 | Amicus Therapeutics Inc | Metodi di potenziamento e/o stabilizzazione della funzione cardiaca in pazienti con malattia di fabry |
| JP7555818B2 (ja) | 2018-02-06 | 2024-09-25 | アミカス セラピューティックス インコーポレイテッド | 妊娠患者におけるファブリー病の治療のためのミガラスタットの使用 |
| HUE064761T2 (hu) | 2018-02-06 | 2024-04-28 | Amicus Therapeutics Inc | Klasszikus FABRY-betegségben szenvedõ betegek kezelése migalasztáttal |
| US20210315875A1 (en) | 2018-08-20 | 2021-10-14 | Amicus Therapeutics, Inc. | Methods of Treating Fabry Disease in Patients Having a Mutation in the GLA Gene |
| US20220313670A1 (en) | 2019-06-11 | 2022-10-06 | Amicus Therapeutics, Inc. | Methods Of Treating Fabry Disease In Patients Having Renal Impairment |
| HRP20260109T1 (hr) | 2019-08-07 | 2026-03-13 | Amicus Therapeutics, Inc. | Migalastat za upotrebu u liječenju fabryjeve bolesti kod pacijenata koji imaju mutaciju u gla genu |
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2007
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