DK3457135T3 - Behandlingsmuligheder til fabrys sygdom - Google Patents
Behandlingsmuligheder til fabrys sygdom Download PDFInfo
- Publication number
- DK3457135T3 DK3457135T3 DK18197135.9T DK18197135T DK3457135T3 DK 3457135 T3 DK3457135 T3 DK 3457135T3 DK 18197135 T DK18197135 T DK 18197135T DK 3457135 T3 DK3457135 T3 DK 3457135T3
- Authority
- DK
- Denmark
- Prior art keywords
- treatment options
- fabrys
- disease
- fabrys 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)
- 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)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Cell Biology (AREA)
- Food Science & Technology (AREA)
- Physics & Mathematics (AREA)
- Pathology (AREA)
- General Physics & Mathematics (AREA)
- Ecology (AREA)
- Biochemistry (AREA)
- Tropical Medicine & Parasitology (AREA)
- Analytical Chemistry (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 | |
| EP15173515.6A EP2955520B1 (en) | 2006-05-16 | 2007-05-16 | Treatment options for fabry disease |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK3457135T3 true DK3457135T3 (da) | 2025-02-24 |
Family
ID=38723987
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK12168451.8T DK2533050T6 (da) | 2006-05-16 | 2007-05-16 | Behandlingsmuligheder ved Fabrys sygdom |
| DK14168748.3T DK2787345T3 (da) | 2006-05-16 | 2007-05-16 | Behandlingsmuligheder for fabrys sygdom |
| DK18197135.9T DK3457135T3 (da) | 2006-05-16 | 2007-05-16 | Behandlingsmuligheder til fabrys sygdom |
Family Applications Before (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK12168451.8T DK2533050T6 (da) | 2006-05-16 | 2007-05-16 | Behandlingsmuligheder ved Fabrys sygdom |
| DK14168748.3T DK2787345T3 (da) | 2006-05-16 | 2007-05-16 | Behandlingsmuligheder for fabrys sygdom |
Country Status (19)
| Country | Link |
|---|---|
| US (9) | US7851143B2 (da) |
| EP (6) | EP4541418A3 (da) |
| JP (7) | JP2009537149A (da) |
| AU (1) | AU2007253900A1 (da) |
| CA (1) | CA2652553C (da) |
| CY (3) | CY1115180T1 (da) |
| DK (3) | DK2533050T6 (da) |
| ES (4) | ES2573498T3 (da) |
| FI (1) | FI3457135T3 (da) |
| HK (1) | HK1218955A1 (da) |
| HU (3) | HUE027569T2 (da) |
| LT (2) | LT3457135T (da) |
| LU (1) | LU93312I2 (da) |
| MX (2) | MX351004B (da) |
| NL (1) | NL300843I2 (da) |
| PL (3) | PL2533050T6 (da) |
| PT (2) | PT3457135T (da) |
| SI (3) | SI3457135T1 (da) |
| WO (1) | WO2007137072A2 (da) |
Families Citing this family (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP4541418A3 (en) | 2006-05-16 | 2025-07-09 | Amicus Therapeutics, Inc. | Assays for diagnosing and evaluating treatment options for fabry disease |
| 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 |
| PL2252313T3 (pl) | 2008-02-12 | 2015-09-30 | Amicus Therapeutics Inc | Sposób przewidywania odpowiedzi na leczenie chorób farmakologicznymi białkami chaperonowymi |
| MX2010009875A (es) * | 2008-03-12 | 2010-11-26 | Amicus Therapeutics Inc | Ensayos para diagnosticar y evaluar opciones de tratamiento para enfermedad de pompe. |
| WO2011063048A2 (en) * | 2009-11-17 | 2011-05-26 | Baylor Research Institute | Urinary triaosylceramide (gb3) as a marker of cardiac disease |
| AU2011223706A1 (en) * | 2010-03-05 | 2012-09-20 | Baylor Research Institute | Involvement of androgen/androgen receptor pathway in Fabry disease |
| EP3698792A1 (en) * | 2011-03-11 | 2020-08-26 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of fabry disease |
| WO2012154967A1 (en) | 2011-05-12 | 2012-11-15 | Proteostasis Therapeutics, Inc. | Proteostasis regulators |
| JP6320931B2 (ja) * | 2011-12-22 | 2018-05-09 | セントジーン アイピー ゲーエムベーハー | 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 |
| JP2015504919A (ja) | 2012-01-25 | 2015-02-16 | プロテオステイシス セラピューティクス,インコーポレイテッド | プロテアソーム活性を調節する化合物 |
| WO2014127038A1 (en) * | 2013-02-12 | 2014-08-21 | Baylor Research Institute | Methods and compositions related to calcium homeostasis and fabry disease |
| JP6546923B2 (ja) * | 2013-12-23 | 2019-07-17 | ビーシーエヌ ペプタイズ エセ.ア.Bcn Peptides S.A. | リソソーム蓄積障害または糖原病の処置における使用のためのエキソサイトーシス活性化化合物としてのビカルタミド類似体または(s)−ビカルタミド |
| 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 |
| US12070453B2 (en) * | 2016-07-19 | 2024-08-27 | Amicus Therapeutics, Inc. | Treatment of Fabry disease in ERT-naïve 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 |
| MX391565B (es) | 2017-05-30 | 2025-03-21 | Amicus Therapeutics Inc | Métodos de tratamiento de pacientes con enfermedad de fabry que tienen insuficiencia renal. |
| TWI795408B (zh) * | 2017-05-30 | 2023-03-11 | 美商阿米庫斯醫療股份有限公司 | 治療有腎損傷的法布里患者的方法 |
| US11923048B1 (en) | 2017-10-03 | 2024-03-05 | Cerner Innovation, Inc. | Determining mucopolysaccharidoses and decision support tool |
| HUE064761T2 (hu) * | 2018-02-06 | 2024-04-28 | Amicus Therapeutics Inc | Klasszikus FABRY-betegségben szenvedõ betegek kezelése migalasztáttal |
| KR20200128676A (ko) | 2018-02-06 | 2020-11-16 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 임신 환자의 파브리병 치료를 위한 미갈라스타트의 용도 |
| AU2019295765B2 (en) | 2018-06-27 | 2024-08-29 | Yumanity, Inc. | Proteasome activity enhancing compounds |
| CN112585130A (zh) | 2018-06-27 | 2021-03-30 | 蛋白质平衡治疗股份有限公司 | 蛋白酶体活性增强化合物 |
| 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 |
| EP3851105A3 (en) | 2019-08-07 | 2021-10-13 | 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 |
|---|---|---|---|---|
| WO1990011353A1 (en) * | 1989-03-24 | 1990-10-04 | Research Corporation Technologies, Inc. | Recombinant alpha-galactosidase, a therapy for fabry disease |
| CA2086413A1 (en) | 1990-06-29 | 1991-12-30 | Yohji Ezure | Piperidine derivatives |
| US5900360A (en) | 1996-04-10 | 1999-05-04 | Welch; William J. | Correction of genetic defects using chemical chaperones |
| 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 |
| ES2531478T3 (es) | 2003-01-31 | 2015-03-16 | The Mount Sinai School Of Medicine Of New York University | Terapia de combinación para tratar trastornos de deficiencia de proteínas |
| 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 |
| CN1901907A (zh) | 2003-11-12 | 2007-01-24 | 阿米库斯治疗学公司 | 用于治疗戈谢病的葡糖并咪唑和多羟基环己烯基胺衍生物 |
| WO2006125141A2 (en) | 2005-05-17 | 2006-11-23 | Amicus Therapeutics, Inc. | A method for the treatment of pompe disease using 1-deoxynojirimycin derivatives |
| ES2537089T3 (es) | 2005-06-08 | 2015-06-02 | Amicus Therapeutics, Inc. | Purificación de imino- y amino-azúcares |
| CA2611011C (en) | 2005-06-08 | 2014-01-28 | Amicus Therapeutics, Inc. | Treatment of cns disorders associated with mutations in genes encoding lysosomal enzymes |
| NZ565953A (en) | 2005-07-27 | 2012-01-12 | Univ Florida | Small compounds that correct protein misfolding and uses thereof |
| EP1982189A4 (en) | 2006-01-27 | 2010-09-08 | Univ Leland Stanford Junior | COMPOSITIONS AND METHODS FOR SCREENING WITH HIGH PASSAGE OF PHARMACOLOGICAL CHAPTERS |
| EP4541418A3 (en) * | 2006-05-16 | 2025-07-09 | Amicus Therapeutics, Inc. | Assays for diagnosing and evaluating treatment options for fabry disease |
| WO2008034628A1 (en) | 2006-09-22 | 2008-03-27 | Krka, Tovarna Zdravil, D.D., Novo Mesto | Aripiprazole hemifumarate and process for its preparation |
| WO2008121826A2 (en) | 2007-03-30 | 2008-10-09 | Amicus Therapeutics, Inc. | Method for the treatment of fabry disease 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 |
| 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. |
| PL2252313T3 (pl) | 2008-02-12 | 2015-09-30 | Amicus Therapeutics Inc | Sposób przewidywania odpowiedzi na leczenie chorób farmakologicznymi białkami chaperonowymi |
| US8321148B2 (en) | 2008-10-24 | 2012-11-27 | Amicus Therapeutics, Inc. | Multiple compartment dosing model |
| US9206457B2 (en) | 2009-05-26 | 2015-12-08 | Amicus Therapeutics, Inc. | Utilization of pharmacological chaperones to improve manufacturing and purification of biologics |
| US20110070643A1 (en) | 2009-05-26 | 2011-03-24 | Do Hung V | Utilization of Pharmacological Chaperones to Improve Manufacturing and Purification of Biologics |
| WO2011063048A2 (en) | 2009-11-17 | 2011-05-26 | Baylor Research Institute | Urinary triaosylceramide (gb3) as a marker of cardiac disease |
| EP3698792A1 (en) | 2011-03-11 | 2020-08-26 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of fabry disease |
| EP2874648A4 (en) | 2012-07-17 | 2015-12-30 | Amicus Therapeutics Inc | CO-FORMULATION OF ALPHA-GALACTOSIDASE A AND 1-DEOXYGALACTONOJIRIMYCIN |
| 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 |
| EP3432882B1 (en) | 2016-03-22 | 2021-09-01 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in patients having the g9331a mutation in the gla gene |
| US12070453B2 (en) | 2016-07-19 | 2024-08-27 | Amicus Therapeutics, Inc. | Treatment of Fabry disease in ERT-naïve and ERT-experienced patients |
| JP2020503900A (ja) | 2017-01-10 | 2020-02-06 | アミカス セラピューティックス インコーポレイテッド | ファブリー病の処置のための組換えアルファ−ガラクトシダーゼa |
| MX391565B (es) | 2017-05-30 | 2025-03-21 | Amicus Therapeutics Inc | Métodos de tratamiento de pacientes con enfermedad de fabry que tienen insuficiencia renal. |
| EA202090242A1 (ru) | 2017-07-19 | 2020-05-26 | Амикус Терапьютикс, Инк. | Лечение болезни фабри у фзт-наивных и фзт-леченных пациентов |
| TWI845479B (zh) | 2017-08-28 | 2024-06-21 | 美商阿米庫斯醫療股份有限公司 | 增強及/或穩定Fabry氏症患者之心臟功能之方法 |
| KR20200128676A (ko) | 2018-02-06 | 2020-11-16 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 임신 환자의 파브리병 치료를 위한 미갈라스타트의 용도 |
| HUE064761T2 (hu) | 2018-02-06 | 2024-04-28 | Amicus Therapeutics Inc | Klasszikus FABRY-betegségben szenvedõ betegek kezelése migalasztáttal |
| CA3110065A1 (en) | 2018-08-20 | 2020-02-27 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in patients having a mutation in the gla gene |
| EA202290024A1 (ru) | 2019-06-11 | 2022-03-14 | Амикус Терапьютикс, Инк. | Способы лечения болезни фабри у пациентов с почечной недостаточностью |
| EP3851105A3 (en) | 2019-08-07 | 2021-10-13 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in patients having a mutation in the gla gene |
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2007
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2010
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2012
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2014
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2015
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2016
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2018
- 2018-04-10 JP JP2018075570A patent/JP6800176B2/ja active Active
- 2018-05-08 US US15/974,222 patent/US10406143B2/en active Active - Reinstated
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2019
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2020
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2022
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2024
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