PL2787345T3 - Możliwości leczenia choroby Fabry’ego - Google Patents
Możliwości leczenia choroby Fabry’egoInfo
- Publication number
- PL2787345T3 PL2787345T3 PL14168748T PL14168748T PL2787345T3 PL 2787345 T3 PL2787345 T3 PL 2787345T3 PL 14168748 T PL14168748 T PL 14168748T PL 14168748 T PL14168748 T PL 14168748T PL 2787345 T3 PL2787345 T3 PL 2787345T3
- Authority
- PL
- Poland
- Prior art keywords
- treatment options
- fabry disease
- fabry
- disease
- options
- Prior art date
Links
- 208000024720 Fabry Disease Diseases 0.000 title 1
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)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Epidemiology (AREA)
- Immunology (AREA)
- Urology & Nephrology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Hematology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Cell Biology (AREA)
- Biotechnology (AREA)
- General Physics & Mathematics (AREA)
- Ecology (AREA)
- Pathology (AREA)
- Tropical Medicine & Parasitology (AREA)
- Biochemistry (AREA)
- Analytical Chemistry (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Food Science & Technology (AREA)
- Cardiology (AREA)
- Heart & Thoracic Surgery (AREA)
- Nutrition Science (AREA)
- Physiology (AREA)
- Obesity (AREA)
- Diabetes (AREA)
- Vascular Medicine (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US80108906P | 2006-05-16 | 2006-05-16 | |
| US85363106P | 2006-10-23 | 2006-10-23 | |
| EP07797506.8A EP2024745B1 (en) | 2006-05-16 | 2007-05-16 | Assays for diagnosing and evaluating treatment options for fabry disease |
| EP14168748.3A EP2787345B8 (en) | 2006-05-16 | 2007-05-16 | Treatment options for fabry disease |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PL2787345T3 true PL2787345T3 (pl) | 2016-08-31 |
Family
ID=38723987
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL18197135.9T PL3457135T3 (pl) | 2006-05-16 | 2007-05-16 | Możliwości leczenia choroby fabry'ego |
| PL14168748T PL2787345T3 (pl) | 2006-05-16 | 2007-05-16 | Możliwości leczenia choroby Fabry’ego |
| PL12168451T PL2533050T6 (pl) | 2006-05-16 | 2007-05-16 | Możliwości leczenia choroby Fabry'ego |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL18197135.9T PL3457135T3 (pl) | 2006-05-16 | 2007-05-16 | Możliwości leczenia choroby fabry'ego |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL12168451T PL2533050T6 (pl) | 2006-05-16 | 2007-05-16 | Możliwości leczenia choroby Fabry'ego |
Country Status (19)
| Country | Link |
|---|---|
| US (9) | US7851143B2 (pl) |
| EP (6) | EP2533050B3 (pl) |
| JP (7) | JP2009537149A (pl) |
| AU (1) | AU2007253900A1 (pl) |
| CA (1) | CA2652553C (pl) |
| CY (3) | CY1115180T1 (pl) |
| DK (3) | DK3457135T3 (pl) |
| ES (4) | ES2573498T3 (pl) |
| FI (1) | FI3457135T3 (pl) |
| HK (1) | HK1218955A1 (pl) |
| HU (3) | HUE027569T2 (pl) |
| LT (2) | LT3457135T (pl) |
| LU (1) | LU93312I2 (pl) |
| MX (2) | MX351004B (pl) |
| NL (1) | NL300843I2 (pl) |
| PL (3) | PL3457135T3 (pl) |
| PT (2) | PT2533050E (pl) |
| SI (3) | SI2533050T1 (pl) |
| WO (1) | WO2007137072A2 (pl) |
Families Citing this family (31)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2009537149A (ja) | 2006-05-16 | 2009-10-29 | アミークス セラピューティックス インコーポレイテッド | ファブリー病に対する治療の選択を診断し、評価するためのアッセイ |
| US9999618B2 (en) | 2007-04-26 | 2018-06-19 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
| WO2008134628A2 (en) * | 2007-04-26 | 2008-11-06 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
| WO2009102895A2 (en) * | 2008-02-12 | 2009-08-20 | Amicus Therapeutics, Inc. | Method to predict response to pharmacological chaperone treatment of diseases |
| WO2009114712A2 (en) * | 2008-03-12 | 2009-09-17 | Amicus Therapeutics, Inc. | Assays for diagnosing and evaluating treatment options for pompe disease |
| AR079057A1 (es) * | 2009-11-17 | 2011-12-21 | Baylor Res Inst | Triaosilceramida urinaria (gb3) como marcador de enfermedad cardiaca |
| AU2011223706A1 (en) * | 2010-03-05 | 2012-09-20 | Baylor Research Institute | Involvement of androgen/androgen receptor pathway in Fabry disease |
| US20140219986A1 (en) * | 2011-03-11 | 2014-08-07 | Amicus Therapeutics ,Inc. | Dosing regimens for the treatment of fabry disease |
| RS58718B1 (sr) | 2011-05-12 | 2019-06-28 | Proteostasis Therapeutics Inc | Regulatori proteostaze |
| ES2864774T3 (es) * | 2011-12-22 | 2021-10-14 | Centogene Ip Gmbh | Combinación de un compuesto que tiene la capacidad de reorganizar una enzima lisosomal y Ambroxol y/o un derivado de Ambroxol |
| WO2013112706A1 (en) | 2012-01-25 | 2013-08-01 | Proteostasis Therapeutics, Inc. | Proteasome activity modulating compounds |
| WO2014127038A1 (en) * | 2013-02-12 | 2014-08-21 | Baylor Research Institute | Methods and compositions related to calcium homeostasis and fabry disease |
| WO2015097088A1 (en) * | 2013-12-23 | 2015-07-02 | Bcn Peptides, S.A. | Bicalutamide analogs or (s)-bi calutam ide as exocytosis activating compounds for use in the treatment of a lysosomal storage disorder or glycogenosis |
| US9675627B2 (en) * | 2014-04-14 | 2017-06-13 | Amicus Therapeutics, Inc. | Dosing regimens for treating and/or preventing cerebral amyloidoses |
| CA2955297C (en) | 2014-07-25 | 2025-07-08 | Takeda Pharmaceutical Company Limited | CRYSTALLINE STRUCTURE OF THE DOMAIN SIMILAR TO A HUMAN FOUR-PHOSPHATE ADAPTER PROTEIN 2 GLYCOLIPID TRANSFER PROTEIN |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| AU2017298376A1 (en) * | 2016-07-19 | 2019-03-07 | Amicus Therapeutics, Inc. | Treatment of Fabry disease in ERT-naive and ERT-experienced patients |
| 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 |
| PT4324522T (pt) * | 2017-05-30 | 2026-03-13 | Amicus Therapeutics Inc | Métodos de tratamento de pacientes com fabry tendo insuficiência renal |
| KR20220116305A (ko) | 2017-05-30 | 2022-08-22 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 신장 손상을 갖는 파브리 환자를 치료하는 방법 |
| US11923048B1 (en) | 2017-10-03 | 2024-03-05 | Cerner Innovation, Inc. | Determining mucopolysaccharidoses and decision support tool |
| EP3749307B1 (en) | 2018-02-06 | 2025-12-10 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
| PT3749308T (pt) * | 2018-02-06 | 2024-01-25 | Amicus Therapeutics Inc | Tratamento de pacientes com doença de fabry clássica com migalastato |
| EP3814336A1 (en) | 2018-06-27 | 2021-05-05 | Proteostasis Therapeutics, Inc. | Proteasome activity enhancing compounds |
| AU2019295765B2 (en) | 2018-06-27 | 2024-08-29 | Yumanity, Inc. | Proteasome activity enhancing compounds |
| 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 |
| EP4289476A3 (en) | 2018-08-20 | 2024-02-21 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in patients having a mutation in the gla gene |
| WO2021026447A1 (en) | 2019-08-07 | 2021-02-11 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in patients having a mutation in the gla gene |
| 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 |
| CA2086413A1 (en) | 1990-06-29 | 1991-12-30 | Yohji Ezure | Piperidine derivatives |
| US6270954B1 (en) | 1996-04-10 | 2001-08-07 | The Regents Of The University Of California | Correction of genetic defects using chemical chaperones |
| US5900360A (en) | 1996-04-10 | 1999-05-04 | Welch; William J. | 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 |
| PT2857036T (pt) | 2003-01-31 | 2017-03-13 | Mount Sinai School Of Medicine Of N Y Univ | Terapia combinada para o tratamento de distúrbios de deficiência proteica |
| US7446098B2 (en) * | 2003-02-18 | 2008-11-04 | Mount Sinai School Of Medicine Of New York University | Combination therapy for treating protein deficiencies |
| WO2004103368A1 (en) | 2003-05-22 | 2004-12-02 | 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 |
| AU2006247065B8 (en) | 2005-05-17 | 2012-07-12 | Amicus Therapeutics, Inc. | A method for the treatment of Pompe disease using 1-deoxynojirimycin derivatives |
| US7973157B2 (en) | 2005-06-08 | 2011-07-05 | Amicus Therapeutics, Inc. | Imino and amino sugar purification |
| MX2007015602A (es) | 2005-06-08 | 2008-02-21 | Amicus Therapeutics Inc | Tratamiento de trastornos del sistema nervioso central asociados con mutaciones en genes que codifican las enzimas lisosomicas. |
| CA2616537A1 (en) | 2005-07-27 | 2007-02-01 | University Of Florida Research Foundation, Inc. | Small compounds that correct protein misfolding and uses thereof |
| WO2007089591A2 (en) | 2006-01-27 | 2007-08-09 | The Board Of Trustees Of The Leland Stanford Junior University | Compositions and methods for high throughput screening of pharmacological chaperones |
| JP2009537149A (ja) | 2006-05-16 | 2009-10-29 | アミークス セラピューティックス インコーポレイテッド | ファブリー病に対する治療の選択を診断し、評価するためのアッセイ |
| UA94624C2 (uk) | 2006-09-22 | 2011-05-25 | Крка, Товарна Здравіл, Д.Д., Ново Мєсто | Арипіпразолу геміфумарат і спосіб його одержання |
| MX2009010557A (es) | 2007-03-30 | 2009-11-19 | Amicus Therapeutics Inc | Metodo para el tratamiento de la enfermedad de fabry que utiliza chaperonas farmacologicas. |
| WO2008134628A2 (en) | 2007-04-26 | 2008-11-06 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
| US9999618B2 (en) | 2007-04-26 | 2018-06-19 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
| WO2009102895A2 (en) | 2008-02-12 | 2009-08-20 | Amicus Therapeutics, Inc. | Method to predict response to pharmacological chaperone treatment of diseases |
| WO2010048532A1 (en) | 2008-10-24 | 2010-04-29 | Amicus Therapeutics, Inc. | Multiple compartment dosing model |
| WO2010138608A1 (en) | 2009-05-26 | 2010-12-02 | Amicus Therapeutics, Inc. | Utilization of pharmacological chaperones to improve manufacturing and purification of biologics |
| US9206457B2 (en) | 2009-05-26 | 2015-12-08 | Amicus Therapeutics, Inc. | Utilization of pharmacological chaperones to improve manufacturing and purification of biologics |
| AR079057A1 (es) | 2009-11-17 | 2011-12-21 | Baylor Res Inst | Triaosilceramida urinaria (gb3) como marcador de enfermedad cardiaca |
| US20140219986A1 (en) | 2011-03-11 | 2014-08-07 | Amicus Therapeutics ,Inc. | Dosing regimens for the treatment of fabry disease |
| 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 |
| HK1209625A1 (en) | 2012-07-17 | 2016-04-08 | Amicus Therapeutics, Inc. | Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation |
| KR20190087673A (ko) | 2016-03-22 | 2019-07-24 | 아미쿠스 세라퓨틱스, 인코포레이티드 | Gla 유전자에 g9331a 돌연변이를 갖는 환자에서 파브리 질병의 치료 방법 |
| AU2017298376A1 (en) | 2016-07-19 | 2019-03-07 | Amicus Therapeutics, Inc. | Treatment of Fabry disease in ERT-naive and ERT-experienced patients |
| TW201829770A (zh) | 2017-01-10 | 2018-08-16 | 美商阿米庫斯醫療股份有限公司 | 用於治療fabry氏病之重組α-半乳糖苷酶A |
| KR20220116305A (ko) | 2017-05-30 | 2022-08-22 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 신장 손상을 갖는 파브리 환자를 치료하는 방법 |
| WO2019017938A1 (en) | 2017-07-19 | 2019-01-24 | Amicus Therapeutics, Inc. | TREATMENT OF FABRY'S DISEASE IN PATIENTS WITHOUT SUBSTITUTED ENZYMATIC THERAPY (TES) AND PATIENTS SUBJECT TO TES |
| PT3675853T (pt) | 2017-08-28 | 2022-11-28 | Amicus Therapeutics Inc | Métodos para melhorar e/ou estabilizar a função cardíaca em pacientes com doença de fabry |
| EP3749307B1 (en) | 2018-02-06 | 2025-12-10 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
| PT3749308T (pt) | 2018-02-06 | 2024-01-25 | Amicus Therapeutics Inc | Tratamento de pacientes com doença de fabry clássica com migalastato |
| EP4289476A3 (en) | 2018-08-20 | 2024-02-21 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in patients having a mutation in the gla gene |
| BR112021024886A2 (pt) | 2019-06-11 | 2022-01-25 | Amicus Therapeutics Inc | Métodos de tratar a doença de fabry em pacientes com insuficiência renal |
| WO2021026447A1 (en) | 2019-08-07 | 2021-02-11 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in patients having a mutation in the gla gene |
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