HUE026543T2 - Eljárás betegségek gyógyászati chaperonnal történõ kezelésére adott válasz elõrejelzésére - Google Patents
Eljárás betegségek gyógyászati chaperonnal történõ kezelésére adott válasz elõrejelzéséreInfo
- Publication number
- HUE026543T2 HUE026543T2 HUE09710178A HUE09710178A HUE026543T2 HU E026543 T2 HUE026543 T2 HU E026543T2 HU E09710178 A HUE09710178 A HU E09710178A HU E09710178 A HUE09710178 A HU E09710178A HU E026543 T2 HUE026543 T2 HU E026543T2
- Authority
- HU
- Hungary
- Prior art keywords
- diseases
- pharmacological chaperone
- predict response
- chaperone treatment
- treatment
- Prior art date
Links
- 108010006519 Molecular Chaperones Proteins 0.000 title 1
- 201000010099 disease Diseases 0.000 title 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title 1
- 230000000144 pharmacologic effect Effects 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
- 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
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/06—Antihyperlipidemics
-
- 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
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
- A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/34—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving hydrolase
-
- 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
- G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
- G01N33/6893—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
-
- 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
- G01N2333/435—Assays involving biological materials from specific organisms or of a specific nature from animals; from humans
- G01N2333/46—Assays involving biological materials from specific organisms or of a specific nature from animals; from humans from vertebrates
- G01N2333/47—Assays involving proteins of known structure or function as defined in the subgroups
-
- 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
- G01N2800/38—Pediatrics
-
- 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)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Hematology (AREA)
- Immunology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Diabetes (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Analytical Chemistry (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Urology & Nephrology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biophysics (AREA)
- Genetics & Genomics (AREA)
- Obesity (AREA)
- Epidemiology (AREA)
- General Engineering & Computer Science (AREA)
- Cell Biology (AREA)
- General Physics & Mathematics (AREA)
- Pathology (AREA)
- Food Science & Technology (AREA)
- Gastroenterology & Hepatology (AREA)
- Endocrinology (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US2814108P | 2008-02-12 | 2008-02-12 | |
| US3568408P | 2008-03-11 | 2008-03-11 | |
| US9363108P | 2008-09-02 | 2008-09-02 | |
| US11349608P | 2008-11-11 | 2008-11-11 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HUE026543T2 true HUE026543T2 (hu) | 2016-06-28 |
Family
ID=40957500
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HUE09710178A HUE026543T2 (hu) | 2008-02-12 | 2009-02-12 | Eljárás betegségek gyógyászati chaperonnal történõ kezelésére adott válasz elõrejelzésére |
| HUE18201960A HUE051377T2 (hu) | 2008-02-12 | 2009-02-12 | Eljárás betegségek gyógyászati chaperonnal történõ kezelésére adott válasz elõrejelzésére |
| HUE15162630A HUE042882T2 (hu) | 2008-02-12 | 2009-02-12 | Eljárás betegségek farmakológiai chaperonnal történõ kezelésére adott válasz elõrejelzésére |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HUE18201960A HUE051377T2 (hu) | 2008-02-12 | 2009-02-12 | Eljárás betegségek gyógyászati chaperonnal történõ kezelésére adott válasz elõrejelzésére |
| HUE15162630A HUE042882T2 (hu) | 2008-02-12 | 2009-02-12 | Eljárás betegségek farmakológiai chaperonnal történõ kezelésére adott válasz elõrejelzésére |
Country Status (16)
| Country | Link |
|---|---|
| US (8) | US8592362B2 (enExample) |
| EP (4) | EP2252313B1 (enExample) |
| JP (7) | JP5844045B2 (enExample) |
| AU (7) | AU2009214648B2 (enExample) |
| CA (1) | CA2715407C (enExample) |
| CY (3) | CY1116466T1 (enExample) |
| DK (3) | DK2946785T3 (enExample) |
| ES (3) | ES2836121T3 (enExample) |
| HR (3) | HRP20150728T1 (enExample) |
| HU (3) | HUE026543T2 (enExample) |
| LT (2) | LT3470077T (enExample) |
| MX (1) | MX2010008835A (enExample) |
| PL (3) | PL3470077T3 (enExample) |
| PT (3) | PT2252313E (enExample) |
| SI (3) | SI2252313T1 (enExample) |
| WO (1) | WO2009102895A2 (enExample) |
Families Citing this family (41)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2533050B3 (en) | 2006-05-16 | 2015-06-24 | Amicus Therapeutics, Inc. | Treatment Options For Fabry Disease |
| 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. |
| AR078498A1 (es) * | 2009-10-01 | 2011-11-09 | Baylor Res Inst | Tratamiento de calvicie de patron masculino por induccion local del defecto metabolico de la enfermedad de fabry |
| US20120283290A1 (en) * | 2011-05-06 | 2012-11-08 | Amicus Therapeutics Inc. | Quantitation of gl3 in urine |
| FI3201320T3 (fi) | 2014-09-30 | 2024-01-08 | Amicus Therapeutics Inc | Erittäin tehokas hapan alfa-glukosidaasi parannetuilla hiilihydraateilla |
| US10172924B2 (en) * | 2015-03-19 | 2019-01-08 | Translate Bio, Inc. | MRNA therapy for pompe disease |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| CA3010205A1 (en) | 2015-12-30 | 2017-07-06 | Amicus Therapeutics, Inc. | Augmented acid alpha-glucosidase for the treatment of pompe disease |
| DK3397273T3 (da) | 2015-12-30 | 2021-08-09 | Amicus Therapeutics Inc | Forstærket syre-alfa-glucosidase til behandling af pompes sygdom |
| 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 |
| SMT202200279T1 (it) | 2016-03-30 | 2022-09-14 | Amicus Therapeutics Inc | Formulazioni comprendenti alfa-glucosidasi acida ricombinante |
| AU2017239641A1 (en) | 2016-03-30 | 2018-10-18 | Amicus Therapeutics, Inc. | Formulations comprising recombinant acid alpha-glucosidase |
| KR20250050123A (ko) * | 2016-03-30 | 2025-04-14 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 고 m6p 재조합 단백질의 선택 방법 |
| CA3031249A1 (en) | 2016-07-19 | 2018-01-25 | Amicus Therapeutics, Inc. | Treatment of fabry disease in ert-naive and ert-experienced patients |
| JP6546135B2 (ja) | 2016-08-24 | 2019-07-17 | 株式会社ノリタケカンパニーリミテド | 積層造形焼成体、積層造形焼成体の製造方法および積層造形焼成体製造用キット |
| JP7042263B2 (ja) * | 2016-10-20 | 2022-03-25 | サンガモ セラピューティクス， インコーポレイテッド | ファブリー病の治療のための方法および組成物 |
| EA202290114A1 (ru) * | 2017-03-30 | 2022-03-29 | Амикус Терапьютикс, Инк. | Способ отбора рекомбинантных белков с высоким содержанием m6p |
| PT3624831T (pt) | 2017-05-15 | 2023-07-04 | Amicus Therapeutics Inc | Alfa-glucosidase ácida humana recombinante |
| EP3630114B1 (en) | 2017-05-30 | 2023-11-01 | Amicus Therapeutics, Inc. | Migalastat for treating fabry patients having renal impairment |
| 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 |
| HUE064761T2 (hu) | 2018-02-06 | 2024-04-28 | Amicus Therapeutics Inc | Klasszikus FABRY-betegségben szenvedõ betegek kezelése migalasztáttal |
| 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 |
| 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 |
| 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 |
| NL2021840B1 (en) | 2018-10-19 | 2020-05-13 | Univ Leiden | Pharmacological Chaperones For Enzyme Treatment Therapy |
| WO2020046132A1 (en) | 2018-08-31 | 2020-03-05 | Leiden University | Pharmacological chaperones for enzyme treatment therapy |
| TW202042812A (zh) | 2019-01-22 | 2020-12-01 | 美商阿米庫斯醫療股份有限公司 | 減少法布瑞氏症患者中之腦血管事件之方法 |
| 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 |
| KR20220058587A (ko) | 2019-09-06 | 2022-05-09 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 생물학적 제제의 포획 및 정제 방법 |
| CA3170718A1 (en) * | 2020-03-06 | 2021-09-10 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in patients having a mutation in the gla gene |
| US11623916B2 (en) | 2020-12-16 | 2023-04-11 | Amicus Therapeutics, Inc. | Highly purified batches of pharmaceutical grade migalastat and methods of producing the same |
| EP4370120A1 (en) | 2021-07-12 | 2024-05-22 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in pediatric patients |
| CN119343147A (zh) | 2022-05-05 | 2025-01-21 | 阿米库斯治疗学公司 | 用于治疗庞贝病的方法 |
| KR20250113510A (ko) | 2022-12-02 | 2025-07-25 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 소아 환자의 후기 발병 폼페병 치료에 대한 방법 |
| TW202436620A (zh) | 2022-12-02 | 2024-09-16 | 美商阿米庫斯醫療股份有限公司 | 用於在兒科患者中治療嬰兒型龐貝氏症之方法 |
| US20240197706A1 (en) | 2022-12-13 | 2024-06-20 | Amicus Therapeutics, Inc. | Methods of improving the pharmacokinetics of migalastat |
| WO2026006625A1 (en) | 2024-06-26 | 2026-01-02 | Amicus Therapeutics, Inc. | Miglustat formulations comprising nitrosamine for use in the treatment of pompe disease |
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| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US270486A (en) * | 1883-01-09 | Bundle-separating attachment for self-binding harvesters | ||
| US203019A (en) * | 1878-04-30 | Improvement in circuits for acoustic or telephonic telegraphs | ||
| US264467A (en) * | 1882-09-19 | Chables h | ||
| US21381A (en) * | 1858-08-31 | Bracelet | ||
| US3586908A (en) | 1969-02-28 | 1971-06-22 | Robert E Vosteen | Automatic potential control system for electrophotography apparatus |
| US5401650A (en) * | 1990-10-24 | 1995-03-28 | The Mount Sinai School Of Medicine Of The City University Of New York | Cloning and expression of biologically active α-galactosidase A |
| US5863903A (en) | 1994-03-09 | 1999-01-26 | Novo Nordisk A/S | Use of hydroxy alkyl piperidine and pyrrolidine compounds to treat diabetes |
| US5844102A (en) | 1994-07-07 | 1998-12-01 | University Of Maryland Baltimore County | Glycohydrolase inhibitors, their preparation and use thereof |
| US6270954B1 (en) | 1996-04-10 | 2001-08-07 | The Regents Of The University Of California | Correction of genetic defects using chemical chaperones |
| EP0959877A4 (en) | 1996-04-10 | 2000-08-23 | Univ California | CORRECTION OF GENETIC ERRORS USING CHEMICAL COMPANIES |
| US5948653A (en) | 1997-03-21 | 1999-09-07 | Pati; Sushma | Sequence alterations using homologous recombination |
| US6274597B1 (en) | 1998-06-01 | 2001-08-14 | Mount Sinai School Of Medicine Of New York University | Method of enhancing lysosomal α-Galactosidase A |
| US6244113B1 (en) | 1999-10-29 | 2001-06-12 | University Of Alabama In Huntsville | Method and apparatus for measuring microgravity acceleration |
| US7842470B2 (en) * | 2001-10-09 | 2010-11-30 | Oregon Health & Science University | Method for pharmacoperones correction of GnRHR mutant protein misfolding |
| EP3782655A1 (en) * | 2005-05-17 | 2021-02-24 | Amicus Therapeutics, Inc. | A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives |
| WO2008045015A1 (en) | 2005-06-08 | 2008-04-17 | Amicus Therapeutics, Inc. | Imino and amino sugar purification |
| WO2006133446A2 (en) * | 2005-06-08 | 2006-12-14 | Amicus Therapeutics, Inc. | Treatment of cns disorders associated with mutations in genes encoding lysosomal enzymes |
| 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 |
| EP2533050B3 (en) | 2006-05-16 | 2015-06-24 | Amicus Therapeutics, Inc. | Treatment Options For Fabry Disease |
| DK1860101T3 (da) | 2006-05-24 | 2011-07-25 | Amicus Therapeutics Inc | Tartrat salt af isofagomin og fremgangsmåder til anvendelse deraf |
| 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 (en) | 2008-10-24 | 2010-04-29 | 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 |
| 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 (en) | 2009-11-17 | 2016-02-10 | Baylor Research Institute | Urinary triaosylceramide (gb3) as a marker of cardiac 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 |
| 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 (en) | 2012-07-17 | 2015-12-30 | Amicus Therapeutics Inc | CO-FORMULATION OF ALPHA-GALACTOSIDASE A AND 1-DEOXYGALACTONOJIRIMYCIN |
| 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 (en) | 2016-07-19 | 2018-01-25 | Amicus Therapeutics, Inc. | Treatment of fabry disease in ert-naive and ert-experienced patients |
| 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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