HUE042882T2 - Eljárás betegségek farmakológiai chaperonnal történõ kezelésére adott válasz elõrejelzésére - Google Patents
Eljárás betegségek farmakológiai chaperonnal történõ kezelésére adott válasz elõrejelzéséreInfo
- Publication number
- HUE042882T2 HUE042882T2 HUE15162630A HUE15162630A HUE042882T2 HU E042882 T2 HUE042882 T2 HU E042882T2 HU E15162630 A HUE15162630 A HU E15162630A HU E15162630 A HUE15162630 A HU E15162630A HU E042882 T2 HUE042882 T2 HU E042882T2
- Authority
- HU
- Hungary
- Prior art keywords
- predicting
- disease
- treating
- response
- pharmacological chaperone
- 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)
- Proteomics, Peptides & Aminoacids (AREA)
- Molecular Biology (AREA)
- Hematology (AREA)
- Immunology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Diabetes (AREA)
- Urology & Nephrology (AREA)
- Analytical Chemistry (AREA)
- Wood Science & Technology (AREA)
- Microbiology (AREA)
- Biomedical Technology (AREA)
- Physics & Mathematics (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Biophysics (AREA)
- Obesity (AREA)
- Epidemiology (AREA)
- Pathology (AREA)
- General Engineering & Computer Science (AREA)
- Food Science & Technology (AREA)
- General Physics & Mathematics (AREA)
- Cell Biology (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 |
|---|---|
| HUE042882T2 true HUE042882T2 (hu) | 2019-07-29 |
Family
ID=40957500
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HUE15162630A HUE042882T2 (hu) | 2008-02-12 | 2009-02-12 | Eljárás betegségek farmakológiai chaperonnal történõ kezelésére adott válasz elõrejelzésére |
| HUE18201960A HUE051377T2 (hu) | 2008-02-12 | 2009-02-12 | Eljárás betegségek gyógyászati chaperonnal történõ kezelésére adott válasz elõrejelzésére |
| HUE09710178A HUE026543T2 (hu) | 2008-02-12 | 2009-02-12 | Eljárás betegségek gyógyászati 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 |
| HUE09710178A HUE026543T2 (hu) | 2008-02-12 | 2009-02-12 | Eljárás betegségek gyógyászati chaperonnal történõ kezelésére adott válasz elõrejelzésére |
Country Status (16)
| Country | Link |
|---|---|
| US (8) | US8592362B2 (enExample) |
| EP (4) | EP2946785B1 (enExample) |
| JP (7) | JP5844045B2 (enExample) |
| AU (7) | AU2009214648B2 (enExample) |
| CA (1) | CA2715407C (enExample) |
| CY (3) | CY1116466T1 (enExample) |
| DK (3) | DK2946785T3 (enExample) |
| ES (3) | ES2541933T3 (enExample) |
| HR (3) | HRP20150728T1 (enExample) |
| HU (3) | HUE042882T2 (enExample) |
| LT (2) | LT2946785T (enExample) |
| MX (1) | MX2010008835A (enExample) |
| PL (3) | PL3470077T3 (enExample) |
| PT (3) | PT3470077T (enExample) |
| SI (3) | SI3470077T1 (enExample) |
| WO (1) | WO2009102895A2 (enExample) |
Families Citing this family (41)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK3457135T3 (da) | 2006-05-16 | 2025-02-24 | Amicus Therapeutics Inc | Behandlingsmuligheder til fabrys sygdom |
| PL3470077T3 (pl) | 2008-02-12 | 2021-04-06 | Amicus Therapeutics, Inc. | Sposób przewidywania odpowiedzi na leczenie chorób chaperonami farmakologicznymi |
| CA2718059A1 (en) * | 2008-03-12 | 2009-09-17 | Amicus Therapeutics, Inc. | Assays for diagnosing and evaluating treatment options for pompe disease |
| 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 |
| HRP20250058T1 (hr) | 2014-09-30 | 2025-03-28 | Amicus Therapeutics, Inc. | Vrlo snažna kisela alfa-glukozidaza s pojačanim ugljikohidratima |
| AU2016233135B2 (en) * | 2015-03-19 | 2021-07-08 | Translate Bio, Inc. | mRNA therapy for pompe disease |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| EA201891507A1 (ru) | 2015-12-30 | 2018-12-28 | Амикус Терапьютикс, Инк. | Кислая альфа-глюкозидаза усиленного действия для лечения болезни помпе |
| LT3957320T (lt) | 2015-12-30 | 2023-12-11 | Amicus Therapeutics, Inc. | Patobulinta rūgštinė alfa-gliukozidazė pompe ligos gydymui |
| US10076514B2 (en) | 2016-03-22 | 2018-09-18 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in patients having the G9331A mutation in the GLA gene |
| TWI813388B (zh) | 2016-03-30 | 2023-08-21 | 美商阿米庫斯醫療股份有限公司 | 包含重組酸性α-葡萄糖苷酶之調配物 |
| IL301319A (en) | 2016-03-30 | 2023-05-01 | Amicus Therapeutics Inc | Formulations that include recombinant acid alpha-glucosidase |
| PE20190127A1 (es) | 2016-03-30 | 2019-01-17 | Amicus Therapeutics Inc | Metodo para seleccionar proteinas recombinantes ricas en m6p |
| TW202408513A (zh) | 2016-07-19 | 2024-03-01 | 美商阿米庫斯醫療股份有限公司 | 治療ert初治的和已經歷ert的患者中之法布瑞氏症 |
| 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 |
| CA3039673A1 (en) * | 2016-10-20 | 2018-04-26 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of fabry disease |
| EA039750B1 (ru) * | 2017-03-30 | 2022-03-10 | Амикус Терапьютикс, Инк. | Способ отбора рекомбинантных белков с высоким содержанием m6p |
| WO2018213340A1 (en) | 2017-05-15 | 2018-11-22 | Amicus Therapeutics, Inc. | Recombinant human acid alpha-glucosidase |
| CA3224537C (en) | 2017-05-30 | 2025-09-09 | Amicus Therapeutics, Inc. | USE OF MIGALASTAT IN THE TREATMENT OF PATIENTS WITH FABRY DISEASE AND RENAL FAILURE |
| EP4327869B1 (en) | 2017-05-30 | 2025-12-10 | Amicus Therapeutics, Inc. | Methods of treating fabry patients having renal impairment |
| EA202090242A1 (ru) | 2017-07-19 | 2020-05-26 | Амикус Терапьютикс, Инк. | Лечение болезни фабри у фзт-наивных и фзт-леченных пациентов |
| KR102790184B1 (ko) | 2017-08-28 | 2025-04-01 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 파브리병을 앓는 환자에서 심장 기능을 강화 및/또는 안정화시키는 방법 |
| AU2019217868A1 (en) | 2018-02-06 | 2020-10-01 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
| AU2019217603A1 (en) | 2018-02-06 | 2020-09-24 | Amicus Therapeutics, Inc. | Treatment of patients with classic Fabry disease |
| 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 |
| TW202112372A (zh) | 2019-06-11 | 2021-04-01 | 美商阿米庫斯醫療股份有限公司 | 治療具有腎損害患者的Fabry氏病之方法 |
| CN114423428A (zh) * | 2019-08-07 | 2022-04-29 | 阿米库斯治疗学公司 | 治疗在gla基因中具有突变的患者的法布里病的方法 |
| US20230220320A1 (en) | 2019-09-06 | 2023-07-13 | Amicus Therapeutics, Inc. | Method For Capturing And Purification Of Biologics |
| EP4114390B1 (en) * | 2020-03-06 | 2024-12-04 | 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 |
| WO2023288210A1 (en) | 2021-07-12 | 2023-01-19 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in pediatric patients |
| WO2023215865A1 (en) | 2022-05-05 | 2023-11-09 | Amicus Therapeutics, Inc. | Methods for treating pompe disease |
| AU2023406510A1 (en) | 2022-12-02 | 2025-07-10 | Amicus Therapeutics, Inc. | Fexamethods for treating infantile-onset pompe disease in pediatric patients |
| CN121057588A (zh) | 2022-12-02 | 2025-12-02 | 阿米库斯治疗学公司 | 用于在儿科患者中治疗迟发性庞贝病的方法 |
| 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 |
|---|---|---|---|---|
| US203019A (en) * | 1878-04-30 | Improvement in circuits for acoustic or telephonic telegraphs | ||
| US264467A (en) * | 1882-09-19 | Chables h | ||
| US270486A (en) * | 1883-01-09 | Bundle-separating attachment for self-binding harvesters | ||
| 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 |
| 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 |
| 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 |
| BRPI0612928A2 (pt) * | 2005-05-17 | 2010-12-07 | Amicus Therapeutics Inc | Método para aumentar a atividade de uma enzima de (alfa)-glucosidade em uma célula e método para tratar doença de pompe |
| CA2611011C (en) * | 2005-06-08 | 2014-01-28 | Amicus Therapeutics, Inc. | Treatment of cns disorders associated with mutations in genes encoding lysosomal enzymes |
| PL2027137T3 (pl) | 2005-06-08 | 2015-08-31 | Amicus Therapeutics Inc | Oczyszczanie imino- i aminocukrów |
| EP1982189A4 (en) * | 2006-01-27 | 2010-09-08 | Univ Leland Stanford Junior | COMPOSITIONS AND METHODS FOR SCREENING WITH HIGH PASSAGE OF PHARMACOLOGICAL CHAPTERS |
| DK3457135T3 (da) | 2006-05-16 | 2025-02-24 | Amicus Therapeutics Inc | Behandlingsmuligheder til fabrys sygdom |
| DE602007013648D1 (de) | 2006-05-24 | 2011-05-19 | Amicus Therapeutics Inc | Tartratsalz aus Isofagomin und Verwendungsverfahren dafür |
| US9999618B2 (en) | 2007-04-26 | 2018-06-19 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
| JP2010525084A (ja) | 2007-04-26 | 2010-07-22 | アミカス セラピューティックス インコーポレイテッド | 薬理シャペロンを用いたリソソーム蓄積症治療のための投薬計画 |
| PL3470077T3 (pl) | 2008-02-12 | 2021-04-06 | Amicus Therapeutics, Inc. | Sposób przewidywania odpowiedzi na leczenie chorób chaperonami farmakologicznymi |
| 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 |
| US20110070643A1 (en) | 2009-05-26 | 2011-03-24 | Do Hung V | 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 |
| CN103974619B (zh) | 2011-03-11 | 2017-02-15 | 阿米库斯治疗学公司 | 治疗法布里病的给药方案 |
| CA2917995C (en) | 2012-07-17 | 2021-01-26 | Amicus Therapeutics, Inc. | Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation |
| 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 |
| US10076514B2 (en) | 2016-03-22 | 2018-09-18 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in patients having the G9331A mutation in the GLA gene |
| TW202408513A (zh) | 2016-07-19 | 2024-03-01 | 美商阿米庫斯醫療股份有限公司 | 治療ert初治的和已經歷ert的患者中之法布瑞氏症 |
| 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 |
| CA3224537C (en) | 2017-05-30 | 2025-09-09 | Amicus Therapeutics, Inc. | USE OF MIGALASTAT IN THE TREATMENT OF PATIENTS WITH FABRY DISEASE AND RENAL FAILURE |
| EA202090242A1 (ru) | 2017-07-19 | 2020-05-26 | Амикус Терапьютикс, Инк. | Лечение болезни фабри у фзт-наивных и фзт-леченных пациентов |
| KR102790184B1 (ko) | 2017-08-28 | 2025-04-01 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 파브리병을 앓는 환자에서 심장 기능을 강화 및/또는 안정화시키는 방법 |
| AU2019217603A1 (en) | 2018-02-06 | 2020-09-24 | Amicus Therapeutics, Inc. | Treatment of patients with classic Fabry disease |
| AU2019217868A1 (en) | 2018-02-06 | 2020-10-01 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
| 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 |
| TW202112372A (zh) | 2019-06-11 | 2021-04-01 | 美商阿米庫斯醫療股份有限公司 | 治療具有腎損害患者的Fabry氏病之方法 |
| CN114423428A (zh) | 2019-08-07 | 2022-04-29 | 阿米库斯治疗学公司 | 治疗在gla基因中具有突变的患者的法布里病的方法 |
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