CN112888439A - 治疗在gla基因中具有突变的患者的法布里病的方法 - Google Patents

治疗在gla基因中具有突变的患者的法布里病的方法 Download PDF

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Publication number
CN112888439A
CN112888439A CN201980069272.8A CN201980069272A CN112888439A CN 112888439 A CN112888439 A CN 112888439A CN 201980069272 A CN201980069272 A CN 201980069272A CN 112888439 A CN112888439 A CN 112888439A
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mutation
patient
migalastat
salt
administered
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埃尔弗里达·本杰明
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Amicus Therapeutics Inc
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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
    • 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

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  • Urology & Nephrology (AREA)
  • Cardiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
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CN201980069272.8A 2018-08-20 2019-01-16 治疗在gla基因中具有突变的患者的法布里病的方法 Pending CN112888439A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862719962P 2018-08-20 2018-08-20
US62/719,962 2018-08-20
PCT/US2019/013761 WO2020040806A1 (en) 2018-08-20 2019-01-16 Methods of treating fabry disease in patients having a mutation in the gla gene

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CN112888439A true CN112888439A (zh) 2021-06-01

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US (2) US12594268B2 (https=)
EP (7) EP4282472A3 (https=)
JP (3) JP2021535107A (https=)
KR (3) KR20240017127A (https=)
CN (1) CN112888439A (https=)
AU (2) AU2019326252A1 (https=)
BR (1) BR112021003137A2 (https=)
CA (1) CA3110065A1 (https=)
CL (3) CL2021000435A1 (https=)
DK (1) DK3840753T3 (https=)
EA (1) EA202190569A1 (https=)
ES (1) ES2993833T3 (https=)
FI (1) FI3840753T3 (https=)
HR (1) HRP20241454T1 (https=)
HU (1) HUE068783T2 (https=)
IL (2) IL325185A (https=)
LT (1) LT3840753T (https=)
MX (2) MX2021002070A (https=)
PL (1) PL3840753T3 (https=)
PT (1) PT3840753T (https=)
RS (1) RS66094B1 (https=)
SG (1) SG11202101117QA (https=)
SI (1) SI3840753T1 (https=)
SM (1) SMT202400436T1 (https=)
WO (1) WO2020040806A1 (https=)

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113969313A (zh) * 2021-11-29 2022-01-25 百世诺(北京)医学检验实验室有限公司 罕见遗传病突变基因及其应用
CN116622750A (zh) * 2023-06-01 2023-08-22 上海勉亦生物科技有限公司 优化的人法布里转基因表达盒及其用途
CN116656704A (zh) * 2023-06-01 2023-08-29 华东理工大学 用于治疗法布里病的转基因表达盒

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ES2573498T3 (es) 2006-05-16 2016-06-08 Amicus Therapeutics, Inc. Opciones de tratamiento para la enfermedad de Fabry
SI3470077T1 (sl) 2008-02-12 2020-12-31 Amicus Therapeutics, Inc. Postopek za napovedovanje odgovora na zdravljenje bolezni s farmakološkim spremljevalcem
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
CA3090496C (en) 2018-02-06 2024-03-26 Amicus Therapeutics, Inc. Use of migalastat for treating fabry disease in pregnant patients
EP4282472A3 (en) 2018-08-20 2024-02-21 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having a mutation in the gla gene
EP3851105B1 (en) 2019-08-07 2026-03-04 Amicus Therapeutics, Inc. Migalastat for use in 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

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US20110152319A1 (en) * 2008-02-12 2011-06-23 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
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WO2014120900A1 (en) * 2013-01-31 2014-08-07 Icahn School Of Medicine At Mount Sinai Enhanced therapeutic regimens for treating fabry disease
TW201733581A (zh) * 2016-03-22 2017-10-01 阿米庫斯醫療股份有限公司 在gla基因中具有g9331a突變的患者中治療法布瑞氏症的方法
WO2018017721A1 (en) * 2016-07-19 2018-01-25 Amicus Therapeutics, Inc. Treatment of fabry disease in ert-naïve and ert-experienced patients
WO2018132471A1 (en) * 2017-01-10 2018-07-19 Amicus Therapeutics, Inc. Recombinant alpha-galactosidase a for treatment of fabry disease

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ES2573498T3 (es) 2006-05-16 2016-06-08 Amicus Therapeutics, Inc. Opciones de tratamiento para la enfermedad de Fabry
AU2008232614A1 (en) 2007-03-30 2008-10-09 Amicus Therapeutics, Inc. Method for the treatment of Fabry disease using pharmacological chaperones
EP2150254A4 (en) 2007-04-26 2010-11-10 Amicus Therapeutics Inc DOSAGES FOR THE TREATMENT OF LYSOSOMAL STORAGE DISEASES WITH PHARMACOLOGICAL CHAPTERONES
US9999618B2 (en) 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
US8321148B2 (en) 2008-10-24 2012-11-27 Amicus Therapeutics, Inc. Multiple compartment dosing model
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JP5739898B2 (ja) 2009-11-17 2015-06-24 ベイラー リサーチ インスティテュートＢａｙｌｏｒ Ｒｅｓｅａｒｃｈ Ｉｎｓｔｉｔｕｔｅ 心疾患マーカーとしての尿中トリアオシルセラミド（ｇｂ３）
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CA2860085A1 (en) 2011-12-22 2013-06-27 Centogene Ip Gmbh Combination of a compound having the ability to rearrange a lysosomal enzyme and ambroxol and/or a derivative of ambroxol
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SMT202400027T1 (it) 2018-02-06 2024-03-13 Amicus Therapeutics Inc Trattamento di pazienti con malattia di fabry classica con migalastat
CA3090496C (en) 2018-02-06 2024-03-26 Amicus Therapeutics, Inc. Use of migalastat for treating fabry disease in pregnant patients
EP4282472A3 (en) 2018-08-20 2024-02-21 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having a mutation in the gla gene
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
AR120055A1 (es) 2019-06-11 2022-02-02 Amicus Therapeutics Inc Métodos para tratar la enfermedad de fabry en pacientes que tienen insuficiencia renal
EP3851105B1 (en) 2019-08-07 2026-03-04 Amicus Therapeutics, Inc. Migalastat for use in treating fabry disease in patients having a mutation in the gla gene
FI4103178T3 (fi) * 2020-02-10 2026-03-23 Amicus Therapeutics Inc Menetelmiä fabryn taudin hoitamiseksi
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

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CN103974619A (zh) * 2011-03-11 2014-08-06 阿米库斯医疗股份有限公司 治疗法布里病的给药方案
WO2014120900A1 (en) * 2013-01-31 2014-08-07 Icahn School Of Medicine At Mount Sinai Enhanced therapeutic regimens for treating fabry disease
TW201733581A (zh) * 2016-03-22 2017-10-01 阿米庫斯醫療股份有限公司 在gla基因中具有g9331a突變的患者中治療法布瑞氏症的方法
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WO2018132471A1 (en) * 2017-01-10 2018-07-19 Amicus Therapeutics, Inc. Recombinant alpha-galactosidase a for treatment of fabry disease

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Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113969313A (zh) * 2021-11-29 2022-01-25 百世诺(北京)医学检验实验室有限公司 罕见遗传病突变基因及其应用
CN116622750A (zh) * 2023-06-01 2023-08-22 上海勉亦生物科技有限公司 优化的人法布里转基因表达盒及其用途
CN116656704A (zh) * 2023-06-01 2023-08-29 华东理工大学 用于治疗法布里病的转基因表达盒
CN116622750B (zh) * 2023-06-01 2024-05-31 上海勉亦生物科技有限公司 优化的人法布里转基因表达盒及其用途
CN116656704B (zh) * 2023-06-01 2024-11-05 华东理工大学 用于治疗法布里病的转基因表达盒

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IL325185A (en) 2026-02-01
CL2024000741A1 (es) 2024-10-04
WO2020040806A1 (en) 2020-02-27
EP3840753A1 (en) 2021-06-30
KR20210046729A (ko) 2021-04-28
ES2993833T3 (en) 2025-01-10
EP4289475A2 (en) 2023-12-13
EP4289476A3 (en) 2024-02-21
AU2019326252A1 (en) 2021-02-25
AU2025205141A1 (en) 2025-07-31
IL280961B1 (en) 2026-01-01
BR112021003137A2 (pt) 2021-05-11
PT3840753T (pt) 2024-10-30
EP4289476A2 (en) 2023-12-13
HUE068783T2 (hu) 2025-01-28
JP2021535107A (ja) 2021-12-16
EP4285990A3 (en) 2024-02-21
EP3840753B1 (en) 2024-07-24
EP4282472A3 (en) 2024-02-21
CL2024000739A1 (es) 2024-10-04
SG11202101117QA (en) 2021-03-30
EP4299117A2 (en) 2024-01-03
US20240277687A1 (en) 2024-08-22
JP2023106398A (ja) 2023-08-01
KR20240017127A (ko) 2024-02-06
US20210315875A1 (en) 2021-10-14
DK3840753T3 (da) 2024-10-28
EP4285991A2 (en) 2023-12-06
EP4289475A3 (en) 2024-02-21
MX2021002070A (es) 2021-07-15
MX2024008350A (es) 2024-07-19
EA202190569A1 (ru) 2021-06-01
SI3840753T1 (sl) 2025-01-31
FI3840753T3 (fi) 2024-10-30
RS66094B1 (sr) 2024-11-29
SMT202400436T1 (it) 2024-11-15
CA3110065A1 (en) 2020-02-27
HRP20241454T1 (hr) 2024-12-20
JP2025165955A (ja) 2025-11-05
CL2021000435A1 (es) 2021-10-22
JP7784398B2 (ja) 2025-12-11
EP4282472A2 (en) 2023-11-29
IL280961A (en) 2021-04-29
EP4299117A3 (en) 2024-02-21
KR20240017128A (ko) 2024-02-06
PL3840753T3 (pl) 2025-01-20
EP4285990A2 (en) 2023-12-06
EP4285991A3 (en) 2024-02-21
US12594268B2 (en) 2026-04-07
LT3840753T (lt) 2024-11-11

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