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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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (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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JP2023071652A Division 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

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JP2021509744A Pending JP2021535107A (ja) 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
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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US12594268B2 (en) 2018-08-20 2026-04-07 Amicus Therapeutics, Inc. Methods of treating Fabry disease in patients having a mutation in the GLA gene
KR20220044560A (ko) 2019-08-07 2022-04-08 아미쿠스 세라퓨틱스, 인코포레이티드 Gla 유전자에 돌연변이를 갖는 환자에서 파브리 질병을 치료하는 방법
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
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
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

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CL2024000741A1 (es) 2024-10-04
SG11202101117QA (en) 2021-03-30
SMT202400436T1 (it) 2024-11-15
IL280961A (en) 2021-04-29
AU2019326252A1 (en) 2021-02-25
US20210315875A1 (en) 2021-10-14
FI3840753T3 (fi) 2024-10-30
EP4299117A3 (en) 2024-02-21
KR20210046729A (ko) 2021-04-28
ES2993833T3 (en) 2025-01-10
MX2024008350A (es) 2024-07-19
CL2024000739A1 (es) 2024-10-04
IL280961B1 (en) 2026-01-01
JP7784398B2 (ja) 2025-12-11
MX2021002070A (es) 2021-07-15
BR112021003137A2 (pt) 2021-05-11
EP4285990A2 (en) 2023-12-06
CN112888439A (zh) 2021-06-01
WO2020040806A1 (en) 2020-02-27
HRP20241454T1 (hr) 2024-12-20
JP2023106398A (ja) 2023-08-01
DK3840753T3 (da) 2024-10-28
JP2025165955A (ja) 2025-11-05
KR20240017128A (ko) 2024-02-06
EP4285991A2 (en) 2023-12-06
EP4289476A3 (en) 2024-02-21
EP4282472A2 (en) 2023-11-29
IL325185A (en) 2026-02-01
EP4289476A2 (en) 2023-12-13
EP4289475A2 (en) 2023-12-13
EP3840753B1 (en) 2024-07-24
KR20240017127A (ko) 2024-02-06
EP4285991A3 (en) 2024-02-21
LT3840753T (lt) 2024-11-11
PT3840753T (pt) 2024-10-30
CA3110065A1 (en) 2020-02-27
EP4289475A3 (en) 2024-02-21
EP4282472A3 (en) 2024-02-21
EP3840753A1 (en) 2021-06-30
US20240277687A1 (en) 2024-08-22
PL3840753T3 (pl) 2025-01-20
US12594268B2 (en) 2026-04-07
EP4299117A2 (en) 2024-01-03
EA202190569A1 (ru) 2021-06-01
CL2021000435A1 (es) 2021-10-22
HUE068783T2 (hu) 2025-01-28
EP4285990A3 (en) 2024-02-21
SI3840753T1 (sl) 2025-01-31
RS66094B1 (sr) 2024-11-29
AU2025205141A1 (en) 2025-07-31

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