MX2010008835A - Metodo para predecir respuesta al tratamiento farmacologico con chaperona de enfermedades. - Google Patents
Metodo para predecir respuesta al tratamiento farmacologico con chaperona de enfermedades.Info
- Publication number
- MX2010008835A MX2010008835A MX2010008835A MX2010008835A MX2010008835A MX 2010008835 A MX2010008835 A MX 2010008835A MX 2010008835 A MX2010008835 A MX 2010008835A MX 2010008835 A MX2010008835 A MX 2010008835A MX 2010008835 A MX2010008835 A MX 2010008835A
- Authority
- MX
- Mexico
- Prior art keywords
- pharmacological chaperone
- diseases
- predict response
- treatment
- galactosidase
- Prior art date
Links
- 238000000034 method Methods 0.000 title abstract 4
- 108010006519 Molecular Chaperones Proteins 0.000 title abstract 3
- 230000000144 pharmacologic effect Effects 0.000 title abstract 3
- 201000010099 disease Diseases 0.000 title 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title 1
- 208000024720 Fabry Disease Diseases 0.000 abstract 2
- 102000002464 Galactosidases Human genes 0.000 abstract 1
- 108010093031 Galactosidases Proteins 0.000 abstract 1
- 208000015439 Lysosomal storage disease Diseases 0.000 abstract 1
- 238000000338 in vitro Methods 0.000 abstract 1
- LXBIFEVIBLOUGU-DPYQTVNSSA-N migalastat Chemical compound OC[C@H]1NC[C@H](O)[C@@H](O)[C@H]1O LXBIFEVIBLOUGU-DPYQTVNSSA-N 0.000 abstract 1
- 229950007469 migalastat Drugs 0.000 abstract 1
- 230000004043 responsiveness Effects 0.000 abstract 1
Classifications
-
- 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)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Diabetes (AREA)
- Zoology (AREA)
- Physics & Mathematics (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Microbiology (AREA)
- Wood Science & Technology (AREA)
- Urology & Nephrology (AREA)
- Analytical Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Biophysics (AREA)
- Genetics & Genomics (AREA)
- Obesity (AREA)
- Epidemiology (AREA)
- Cell Biology (AREA)
- General Engineering & Computer Science (AREA)
- Food Science & Technology (AREA)
- General Physics & Mathematics (AREA)
- Pathology (AREA)
- Gastroenterology & Hepatology (AREA)
- Emergency Medicine (AREA)
Applications Claiming Priority (5)
| 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 | |
| PCT/US2009/033963 WO2009102895A2 (en) | 2008-02-12 | 2009-02-12 | Method to predict response to pharmacological chaperone treatment of diseases |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2010008835A true MX2010008835A (es) | 2010-10-20 |
Family
ID=40957500
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2010008835A MX2010008835A (es) | 2008-02-12 | 2009-02-12 | Metodo para predecir respuesta al tratamiento farmacologico con chaperona de enfermedades. |
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) | DK2252313T3 (enExample) |
| ES (3) | ES2836121T3 (enExample) |
| HR (3) | HRP20150728T1 (enExample) |
| HU (3) | HUE042882T2 (enExample) |
| LT (2) | LT2946785T (enExample) |
| MX (1) | MX2010008835A (enExample) |
| PL (3) | PL2946785T3 (enExample) |
| PT (3) | PT2946785T (enExample) |
| SI (3) | SI2946785T1 (enExample) |
| WO (1) | WO2009102895A2 (enExample) |
Families Citing this family (44)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX351004B (es) | 2006-05-16 | 2017-09-26 | Nat Institutes Of Health | Ensayos para diagnosticar y evaluar opciones de tratamiento para enfermedad de fabry. |
| WO2009102895A2 (en) | 2008-02-12 | 2009-08-20 | Amicus Therapeutics, Inc. | Method to predict response to pharmacological chaperone treatment of diseases |
| AU2009223125A1 (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 |
| JP6851964B2 (ja) | 2014-09-30 | 2021-03-31 | アミカス セラピューティックス インコーポレイテッド | 炭水化物が増大している非常に強力な酸性アルファ−グルコシダーゼ |
| EP3270894B1 (en) * | 2015-03-19 | 2021-02-24 | Translate Bio, Inc. | Mrna therapy for pompe disease |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| ES3063549T3 (en) | 2015-12-30 | 2026-04-17 | Amicus Therapeutics Inc | Augmented acid alpha-glucosidase for the treatment of pompe disease |
| IL299470B2 (en) | 2015-12-30 | 2026-05-01 | Amicus Therapeutics Inc | Improved acid alpha-glucosidase for the treatment of Pompe 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 |
| CA3019354A1 (en) | 2016-03-30 | 2017-10-05 | Amicus Therapeutics, Inc. | Method for selection of high m6p recombinant proteins |
| PL3436053T3 (pl) | 2016-03-30 | 2022-08-01 | Amicus Therapeutics, Inc. | Formulacje zawierające rekombinowaną kwaśną alfa-glukozydazę |
| KR102618519B1 (ko) | 2016-03-30 | 2023-12-28 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 재조합 산 알파-글루코시다제를 포함하는 제형 |
| WO2018017721A1 (en) | 2016-07-19 | 2018-01-25 | Amicus Therapeutics, Inc. | Treatment of fabry disease in ert-naïve 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 |
| KR102712926B1 (ko) * | 2016-10-20 | 2024-10-07 | 상가모 테라퓨틱스, 인코포레이티드 | 파브리병의 치료를 위한 방법 및 조성물 |
| EA039750B1 (ru) * | 2017-03-30 | 2022-03-10 | Амикус Терапьютикс, Инк. | Способ отбора рекомбинантных белков с высоким содержанием m6p |
| CN119113091A (zh) | 2017-05-15 | 2024-12-13 | 阿米库斯治疗学公司 | 重组人类酸性α-葡萄糖苷酶 |
| AU2018277756A1 (en) | 2017-05-30 | 2020-01-23 | Amicus Therapeutics, Inc. | Methods of treating fabry patients having renal impairment |
| PL4062916T3 (pl) | 2017-05-30 | 2025-03-17 | Amicus Therapeutics, Inc. | Leczenie pacjentów z chorobą Fabry’ego mających pewne mutacje |
| EA202090242A1 (ru) | 2017-07-19 | 2020-05-26 | Амикус Терапьютикс, Инк. | Лечение болезни фабри у фзт-наивных и фзт-леченных пациентов |
| WO2019046244A1 (en) | 2017-08-28 | 2019-03-07 | Amicus Therapeutics, Inc. | METHODS FOR ENHANCING AND / OR STABILIZING CARDIAC FUNCTION IN PATIENTS WITH FABRY DISEASE |
| EP4717319A3 (en) | 2018-02-06 | 2026-06-10 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in pregnant patients |
| AU2019217603A1 (en) | 2018-02-06 | 2020-09-24 | Amicus Therapeutics, Inc. | Treatment of patients with classic Fabry disease |
| WO2020023390A1 (en) | 2018-07-25 | 2020-01-30 | Modernatx, Inc. | Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders |
| LT3840753T (lt) | 2018-08-20 | 2024-11-11 | Amicus Therapeutics, Inc. | Migalastatas skirtas naudoti fabry ligos gydymo būdui, pacientams turintiems gla geno mutaciją |
| 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 |
| CN113423419A (zh) | 2019-01-04 | 2021-09-21 | 桑格摩生物治疗股份有限公司 | 用于治疗法布里病的方法和组合物 |
| 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 |
| KR20220044560A (ko) * | 2019-08-07 | 2022-04-08 | 아미쿠스 세라퓨틱스, 인코포레이티드 | Gla 유전자에 돌연변이를 갖는 환자에서 파브리 질병을 치료하는 방법 |
| BR112022004000A2 (pt) | 2019-09-06 | 2022-05-31 | Amicus Therapeutics Inc | Método para captura e purificação de biológicas |
| HRP20260399T1 (hr) | 2020-02-10 | 2026-05-08 | Amicus Therapeutics, Inc. | Postupci liječenja fabryjeve bolesti |
| FI4114390T3 (fi) * | 2020-03-06 | 2025-03-07 | Amicus Therapeutics Inc | Menetelmiä fabryn taudin hoitamiseksi potilaissa, joilla on mutaatio gla-geenissä |
| JP2023531508A (ja) | 2020-06-23 | 2023-07-24 | ヴィルテック バイオ，インク． | 出血性ショックを処置するための組成物および方法 |
| US11623916B2 (en) | 2020-12-16 | 2023-04-11 | Amicus Therapeutics, Inc. | Highly purified batches of pharmaceutical grade migalastat and methods of producing the same |
| JP2024525760A (ja) | 2021-07-12 | 2024-07-12 | アミカス セラピューティックス インコーポレイテッド | 小児患者のファブリー病を治療する方法 |
| WO2023215865A1 (en) | 2022-05-05 | 2023-11-09 | Amicus Therapeutics, Inc. | Methods for treating pompe disease |
| AR131256A1 (es) | 2022-12-02 | 2025-03-05 | Amicus Therapeutics Inc | Métodos para tratar la enfermedad de pompe de inicio tardío en pacientes pediátricos |
| WO2024119091A1 (en) | 2022-12-02 | 2024-06-06 | Amicus Therapeutics, Inc. | Fexamethods for treating infantile-onset pompe disease in pediatric patients |
| 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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| 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 |
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| 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 |
| MX351004B (es) | 2006-05-16 | 2017-09-26 | Nat Institutes Of Health | Ensayos para diagnosticar y evaluar opciones de tratamiento para enfermedad de fabry. |
| WO2007140212A2 (en) | 2006-05-24 | 2007-12-06 | Amicus Therapeutics, Inc. | Tartrate salt of isofagomine and methods of use |
| AU2008245578A1 (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 |
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| EP2502068B1 (en) | 2009-11-17 | 2016-02-10 | Baylor Research Institute | Urinary triaosylceramide (gb3) as a marker of cardiac disease |
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| EP2874648A4 (en) | 2012-07-17 | 2015-12-30 | Amicus Therapeutics Inc | ALPHA GALACTOSIDASE A AND 1-DEOXYGALACTONO JIRIMYCIN CO FORMULATION |
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| 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 |
| WO2018017721A1 (en) | 2016-07-19 | 2018-01-25 | Amicus Therapeutics, Inc. | Treatment of fabry disease in ert-naïve and ert-experienced patients |
| US11357834B2 (en) | 2017-01-10 | 2022-06-14 | Amicus Therapeutics, Inc. | Recombinant α-galactosidase A for treatment of Fabry disease |
| AU2018277756A1 (en) | 2017-05-30 | 2020-01-23 | 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 |
| WO2019046244A1 (en) | 2017-08-28 | 2019-03-07 | Amicus Therapeutics, Inc. | METHODS FOR ENHANCING AND / OR STABILIZING CARDIAC FUNCTION IN PATIENTS WITH FABRY DISEASE |
| AU2019217603A1 (en) | 2018-02-06 | 2020-09-24 | Amicus Therapeutics, Inc. | Treatment of patients with classic Fabry disease |
| EP4717319A3 (en) | 2018-02-06 | 2026-06-10 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in pregnant patients |
| LT3840753T (lt) | 2018-08-20 | 2024-11-11 | Amicus Therapeutics, Inc. | Migalastatas skirtas naudoti fabry ligos gydymo būdui, pacientams turintiems gla geno mutaciją |
| US20220313670A1 (en) | 2019-06-11 | 2022-10-06 | Amicus Therapeutics, Inc. | Methods Of Treating Fabry Disease In Patients Having Renal Impairment |
| KR20220044560A (ko) | 2019-08-07 | 2022-04-08 | 아미쿠스 세라퓨틱스, 인코포레이티드 | Gla 유전자에 돌연변이를 갖는 환자에서 파브리 질병을 치료하는 방법 |
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