JP5844045B2 - 疾病の薬理シャペロン治療に対する応答性を予測する方法 - Google Patents

疾病の薬理シャペロン治療に対する応答性を予測する方法 Download PDF

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JP5844045B2
JP5844045B2 JP2010546901A JP2010546901A JP5844045B2 JP 5844045 B2 JP5844045 B2 JP 5844045B2 JP 2010546901 A JP2010546901 A JP 2010546901A JP 2010546901 A JP2010546901 A JP 2010546901A JP 5844045 B2 JP5844045 B2 JP 5844045B2
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JP2011514152A (ja
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エルフリダ・ベンジャミン
ブイ・ハング・ド
ウ・シャオヤン
ジョン・フラナガン
ブランドン・ウストマン
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アミカス セラピューティックス インコーポレイテッド
アミカス セラピューティックス インコーポレイテッド
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    • A—HUMAN NECESSITIES
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    • 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
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    • A—HUMAN NECESSITIES
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    • 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
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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

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JP2010546901A 2008-02-12 2009-02-12 疾病の薬理シャペロン治療に対する応答性を予測する方法 Active JP5844045B2 (ja)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US2814108P 2008-02-12 2008-02-12
US61/028,141 2008-02-12
US3568408P 2008-03-11 2008-03-11
US61/035,684 2008-03-11
US9363108P 2008-09-02 2008-09-02
US61/093,631 2008-09-02
US11349608P 2008-11-11 2008-11-11
US61/113,496 2008-11-11
PCT/US2009/033963 WO2009102895A2 (en) 2008-02-12 2009-02-12 Method to predict response to pharmacological chaperone treatment of diseases

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JP5844045B2 true JP5844045B2 (ja) 2016-01-13

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JP2010546901A Active JP5844045B2 (ja) 2008-02-12 2009-02-12 疾病の薬理シャペロン治療に対する応答性を予測する方法
JP2014244283A Pending JP2015091239A (ja) 2008-02-12 2014-12-02 疾病の薬理シャペロン治療に対する応答性を予測する方法
JP2016039171A Active JP6672013B2 (ja) 2008-02-12 2016-03-01 疾病の薬理シャペロン治療に対する応答性を予測する方法
JP2018245609A Active JP6837469B2 (ja) 2008-02-12 2018-12-27 疾病の薬理シャペロン治療に対する応答性を予測する方法
JP2021019383A Active JP7277493B2 (ja) 2008-02-12 2021-02-09 疾病の薬理シャペロン治療に対する応答性を予測する方法
JP2023076515A Withdrawn JP2023109807A (ja) 2008-02-12 2023-05-08 疾病の薬理シャペロン治療に対する応答性を予測する方法
JP2025097940A Pending JP2025148351A (ja) 2008-02-12 2025-06-11 疾病の薬理シャペロン治療に対する応答性を予測する方法

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JP2014244283A Pending JP2015091239A (ja) 2008-02-12 2014-12-02 疾病の薬理シャペロン治療に対する応答性を予測する方法
JP2016039171A Active JP6672013B2 (ja) 2008-02-12 2016-03-01 疾病の薬理シャペロン治療に対する応答性を予測する方法
JP2018245609A Active JP6837469B2 (ja) 2008-02-12 2018-12-27 疾病の薬理シャペロン治療に対する応答性を予測する方法
JP2021019383A Active JP7277493B2 (ja) 2008-02-12 2021-02-09 疾病の薬理シャペロン治療に対する応答性を予測する方法
JP2023076515A Withdrawn JP2023109807A (ja) 2008-02-12 2023-05-08 疾病の薬理シャペロン治療に対する応答性を予測する方法
JP2025097940A Pending JP2025148351A (ja) 2008-02-12 2025-06-11 疾病の薬理シャペロン治療に対する応答性を予測する方法

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US (8) US8592362B2 (cg-RX-API-DMAC7.html)
EP (4) EP3470077B1 (cg-RX-API-DMAC7.html)
JP (7) JP5844045B2 (cg-RX-API-DMAC7.html)
AU (7) AU2009214648B2 (cg-RX-API-DMAC7.html)
CA (1) CA2715407C (cg-RX-API-DMAC7.html)
CY (3) CY1116466T1 (cg-RX-API-DMAC7.html)
DK (3) DK3470077T3 (cg-RX-API-DMAC7.html)
ES (3) ES2836121T3 (cg-RX-API-DMAC7.html)
HR (3) HRP20150728T1 (cg-RX-API-DMAC7.html)
HU (3) HUE042882T2 (cg-RX-API-DMAC7.html)
LT (2) LT3470077T (cg-RX-API-DMAC7.html)
MX (1) MX2010008835A (cg-RX-API-DMAC7.html)
PL (3) PL2252313T3 (cg-RX-API-DMAC7.html)
PT (3) PT2946785T (cg-RX-API-DMAC7.html)
SI (3) SI2252313T1 (cg-RX-API-DMAC7.html)
WO (1) WO2009102895A2 (cg-RX-API-DMAC7.html)

Families Citing this family (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2533050T6 (da) 2006-05-16 2015-09-21 Amicus Therapeutics Inc Behandlingsmuligheder ved Fabrys sygdom
HUE042882T2 (hu) 2008-02-12 2019-07-29 Amicus Therapeutics Inc Eljárás betegségek farmakológiai chaperonnal történõ kezelésére adott válasz elõrejelzésére
JP2011517556A (ja) * 2008-03-12 2011-06-16 アミカス セラピューティックス インコーポレイテッド ポンペ病のための治療選択肢を診断し評価するための検定
WO2011041718A2 (en) * 2009-10-01 2011-04-07 Baylor Research Institute Treatment of male-pattern baldness by local induction of the metabolic defect of fabry disease
US20120283290A1 (en) * 2011-05-06 2012-11-08 Amicus Therapeutics Inc. Quantitation of gl3 in urine
CA2961762C (en) 2014-09-30 2024-03-05 Amicus Therapeutics, Inc. Highly potent acid alpha-glucosidase with enhanced carbohydrates
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
DK3957320T5 (da) 2015-12-30 2024-08-05 Amicus Therapeutics Inc Forstærket syre-alfa-glucosidase til behandling af pompes sygdom
MY198085A (en) 2015-12-30 2023-07-31 Amicus Therapeutics Inc Augmented 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
US10227577B2 (en) * 2016-03-30 2019-03-12 Amicus Therapeutics, Inc. Method for selection of high M6P recombinant proteins
LT3436053T (lt) 2016-03-30 2022-07-11 Amicus Therapeutics, Inc. Kompozicijos, apimančios rekombinantinę rūgštinę alfa-gliukozidazę
KR102618519B1 (ko) 2016-03-30 2023-12-28 아미쿠스 세라퓨틱스, 인코포레이티드 재조합 산 알파-글루코시다제를 포함하는 제형
KR20240017126A (ko) 2016-07-19 2024-02-06 아미쿠스 세라퓨틱스, 인코포레이티드 Ert-치료경험이 없는 환자 및 있는 환자에서 파브리병의 치료
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
EP3528852B1 (en) * 2016-10-20 2025-09-03 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
HRP20230576T1 (hr) 2017-05-15 2023-09-01 Amicus Therapeutics, Inc. Rekombinantna ljudska kisela alfa-glukozidaza
BR112019025083B1 (pt) 2017-05-30 2022-08-09 Amicus Therapeutics, Inc Métodos de tratamento de pacientes portadores de fabry com insuficiência renal
HUE069837T2 (hu) 2017-05-30 2025-04-28 Amicus Therapeutics Inc Bizonyos mutációkat hordozó Fabry-kóros betegek kezelése
SG11202000423XA (en) 2017-07-19 2020-02-27 Amicus Therapeutics Inc Treatment of fabry disease in ert-naïve and ert-experienced patients
IL310648A (en) 2017-08-28 2024-04-01 Amicus Therapeutics Inc Methods for increasing and/or stabilizing cardiac functions in patients with Fabry disease
KR20200128675A (ko) 2018-02-06 2020-11-16 아미쿠스 세라퓨틱스, 인코포레이티드 전형적 파브리병 환자의 치료
EP3749307B1 (en) 2018-02-06 2025-12-10 Amicus Therapeutics, Inc. Use of migalastat for treating fabry disease in pregnant patients
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
EP4299117A3 (en) 2018-08-20 2024-02-21 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
EA202290024A1 (ru) 2019-06-11 2022-03-14 Амикус Терапьютикс, Инк. Способы лечения болезни фабри у пациентов с почечной недостаточностью
CA3147055A1 (en) 2019-08-07 2021-02-11 Elfrida Benjamin Methods of treating fabry disease in patients having a mutation in the gla gene
WO2021046443A1 (en) 2019-09-06 2021-03-11 Amicus Therapeutics, Inc. Method for capturing and purification of biologics
US20230136297A1 (en) * 2020-03-06 2023-05-04 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
KR20240034203A (ko) 2021-07-12 2024-03-13 아미쿠스 세라퓨틱스, 인코포레이티드 소아청소년 환자에서 파브리병을 치료하는 방법
JP2025515159A (ja) 2022-05-05 2025-05-13 アミカス セラピューティックス インコーポレイテッド ポンペ病の治療方法
AU2023406479A1 (en) 2022-12-02 2025-07-03 Amicus Therapeutics, Inc. Methods for treating late onset pompe disease in pediatric patients
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

Family Cites Families (41)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US21381A (en) * 1858-08-31 Bracelet
US203019A (en) * 1878-04-30 Improvement in circuits for acoustic or telephonic telegraphs
US270486A (en) * 1883-01-09 Bundle-separating attachment for self-binding harvesters
US264467A (en) * 1882-09-19 Chables h
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
JP2000509971A (ja) 1996-04-10 2000-08-08 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 化学的シャペローンを用いる遺伝的欠陥の修正
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
MX2007014470A (es) * 2005-05-17 2008-02-06 Amicus Therapeutics Inc Metodo para el tratamiento de enfermedad de pompe usando derivados de 1-desoxinojirimicina.
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.
PL2027137T3 (pl) 2005-06-08 2015-08-31 Amicus Therapeutics Inc Oczyszczanie imino- i aminocukrów
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
DK2533050T6 (da) * 2006-05-16 2015-09-21 Amicus Therapeutics Inc Behandlingsmuligheder ved 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
US9056101B2 (en) 2007-04-26 2015-06-16 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
HUE042882T2 (hu) 2008-02-12 2019-07-29 Amicus Therapeutics Inc Eljárás betegségek farmakológiai chaperonnal történõ kezelésére adott válasz elõrejelzésére
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
CN104164412A (zh) 2009-05-26 2014-11-26 阿米库斯治疗学公司 利用药物分子伴侣改善生物制剂的生产和纯化
WO2011063048A2 (en) 2009-11-17 2011-05-26 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
US9694056B2 (en) 2012-07-17 2017-07-04 Amicus Therapeutics, Inc. α-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
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
KR20240017126A (ko) 2016-07-19 2024-02-06 아미쿠스 세라퓨틱스, 인코포레이티드 Ert-치료경험이 없는 환자 및 있는 환자에서 파브리병의 치료
EP3568152A1 (en) 2017-01-10 2019-11-20 Amicus Therapeutics, Inc. Recombinant alpha-galactosidase a for treatment of fabry disease
BR112019025083B1 (pt) 2017-05-30 2022-08-09 Amicus Therapeutics, Inc Métodos de tratamento de pacientes portadores de fabry com insuficiência renal
SG11202000423XA (en) 2017-07-19 2020-02-27 Amicus Therapeutics Inc Treatment of fabry disease in ert-naïve and ert-experienced patients
IL310648A (en) 2017-08-28 2024-04-01 Amicus Therapeutics Inc Methods for increasing and/or stabilizing cardiac functions in patients with Fabry disease
EP3749307B1 (en) 2018-02-06 2025-12-10 Amicus Therapeutics, Inc. Use of migalastat for treating fabry disease in pregnant patients
KR20200128675A (ko) 2018-02-06 2020-11-16 아미쿠스 세라퓨틱스, 인코포레이티드 전형적 파브리병 환자의 치료
EP4299117A3 (en) 2018-08-20 2024-02-21 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
CA3147055A1 (en) 2019-08-07 2021-02-11 Elfrida Benjamin Methods of treating fabry disease in patients having a mutation in the gla gene

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