JP2021535107A - Ｇｌａ遺伝子の突然変異を有する患者におけるファブリー病を治療する方法 - Google Patents

Ｇｌａ遺伝子の突然変異を有する患者におけるファブリー病を治療する方法 Download PDF

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JP2021535107A
JP2021535107A JP2021509744A JP2021509744A JP2021535107A JP 2021535107 A JP2021535107 A JP 2021535107A JP 2021509744 A JP2021509744 A JP 2021509744A JP 2021509744 A JP2021509744 A JP 2021509744A JP 2021535107 A JP2021535107 A JP 2021535107A
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migalastat
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エルフリダ ベンジャミン，
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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
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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)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Wood Science & Technology (AREA)
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  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
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JP2021509744A 2018-08-20 2019-01-16 Ｇｌａ遺伝子の突然変異を有する患者におけるファブリー病を治療する方法 Pending JP2021535107A (ja)

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JP2023071652A JP7784398B2 (ja) 2018-08-20 2023-04-25 Ｇｌａ遺伝子の突然変異を有する患者におけるファブリー病を治療する方法
JP2025117816A JP2025165955A (ja) 2018-08-20 2025-07-14 Ｇｌａ遺伝子の突然変異を有する患者におけるファブリー病を治療する方法

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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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JP2021535107A5 JP2021535107A5 (https=) 2022-02-24
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JP2023071652A Active JP7784398B2 (ja) 2018-08-20 2023-04-25 Ｇｌａ遺伝子の突然変異を有する患者におけるファブリー病を治療する方法
JP2025117816A Pending JP2025165955A (ja) 2018-08-20 2025-07-14 Ｇｌａ遺伝子の突然変異を有する患者におけるファブリー病を治療する方法

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JP2025117816A Pending JP2025165955A (ja) 2018-08-20 2025-07-14 Ｇｌａ遺伝子の突然変異を有する患者におけるファブリー病を治療する方法

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EP (7) EP4282472A3 (https=)
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KR (3) KR20240017127A (https=)
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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
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
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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ES2573498T3 (es) 2006-05-16 2016-06-08 Amicus Therapeutics, Inc. Opciones de tratamiento para la enfermedad de Fabry
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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
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
CN112888439A (zh) 2021-06-01
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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