JP7680356B2 - ファブリー病患者の脳血管事象のリスク低減におけるミガロスタットの使用 - Google Patents

ファブリー病患者の脳血管事象のリスク低減におけるミガロスタットの使用 Download PDF

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JP7680356B2
JP7680356B2 JP2021542102A JP2021542102A JP7680356B2 JP 7680356 B2 JP7680356 B2 JP 7680356B2 JP 2021542102 A JP2021542102 A JP 2021542102A JP 2021542102 A JP2021542102 A JP 2021542102A JP 7680356 B2 JP7680356 B2 JP 7680356B2
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migalastat
patient
salt
cbv
gal
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JP2022518733A5 (https=
JP2022518733A (ja
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ニーナ スクバン，
ヒャルマー ラガスト，
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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
    • A61P25/00—Drugs for disorders of the nervous system
    • 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
    • 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
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis

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  • Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Epidemiology (AREA)
  • Urology & Nephrology (AREA)
  • Vascular Medicine (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
JP2021542102A 2019-01-22 2020-01-22 ファブリー病患者の脳血管事象のリスク低減におけるミガロスタットの使用 Active JP7680356B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025077689A JP2025128105A (ja) 2019-01-22 2025-05-08 ファブリー病患者の脳血管事象のリスク低減におけるミガロスタットの使用

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962795524P 2019-01-22 2019-01-22
US62/795,524 2019-01-22
PCT/US2020/014531 WO2020154354A1 (en) 2019-01-22 2020-01-22 Use of migalostat in reducing the risk of cerebrovascular event in patients with fabry disease

Related Child Applications (1)

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JP2025077689A Division JP2025128105A (ja) 2019-01-22 2025-05-08 ファブリー病患者の脳血管事象のリスク低減におけるミガロスタットの使用

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JP2022518733A JP2022518733A (ja) 2022-03-16
JP2022518733A5 JP2022518733A5 (https=) 2023-01-30
JP7680356B2 true JP7680356B2 (ja) 2025-05-20

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JP2021542102A Active JP7680356B2 (ja) 2019-01-22 2020-01-22 ファブリー病患者の脳血管事象のリスク低減におけるミガロスタットの使用
JP2025077689A Pending JP2025128105A (ja) 2019-01-22 2025-05-08 ファブリー病患者の脳血管事象のリスク低減におけるミガロスタットの使用

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JP2025077689A Pending JP2025128105A (ja) 2019-01-22 2025-05-08 ファブリー病患者の脳血管事象のリスク低減におけるミガロスタットの使用

Country Status (17)

Country Link
US (1) US20220087993A1 (https=)
EP (2) EP4523750A3 (https=)
JP (2) JP7680356B2 (https=)
AR (1) AR117861A1 (https=)
DK (1) DK3914247T3 (https=)
ES (1) ES3007212T3 (https=)
FI (1) FI3914247T3 (https=)
HR (1) HRP20250078T1 (https=)
HU (1) HUE069934T2 (https=)
LT (1) LT3914247T (https=)
PL (1) PL3914247T3 (https=)
PT (1) PT3914247T (https=)
RS (1) RS66410B1 (https=)
SI (1) SI3914247T1 (https=)
SM (1) SMT202500034T1 (https=)
TW (1) TW202042812A (https=)
WO (1) WO2020154354A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6274597B1 (en) 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
MX2009010557A (es) 2007-03-30 2009-11-19 Amicus Therapeutics Inc Metodo para el tratamiento de la enfermedad de fabry que utiliza chaperonas farmacologicas.
US9999618B2 (en) * 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
EP2946785B1 (en) 2008-02-12 2018-10-24 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
TW202408513A (zh) * 2016-07-19 2024-03-01 美商阿米庫斯醫療股份有限公司 治療ert初治的和已經歷ert的患者中之法布瑞氏症

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Gere Sunder-Plassmann et al.，Expert Opinion on Prphan Drugs，2018年，Vol. 6, No. 5，p.301-309
Hughes DA et al.，J Med Genet，2017年，Vol. 54，p. 288-296

Also Published As

Publication number Publication date
PT3914247T (pt) 2025-01-23
PL3914247T3 (pl) 2025-04-14
EP3914247B1 (en) 2024-11-13
ES3007212T3 (en) 2025-03-19
FI3914247T3 (fi) 2025-02-16
RS66410B1 (sr) 2025-02-28
LT3914247T (lt) 2025-01-27
SI3914247T1 (sl) 2025-03-31
HRP20250078T1 (hr) 2025-03-28
DK3914247T3 (da) 2025-01-27
SMT202500034T1 (it) 2025-03-12
HUE069934T2 (hu) 2025-04-28
EP3914247A1 (en) 2021-12-01
WO2020154354A1 (en) 2020-07-30
JP2022518733A (ja) 2022-03-16
EP4523750A3 (en) 2025-06-11
JP2025128105A (ja) 2025-09-02
US20220087993A1 (en) 2022-03-24
TW202042812A (zh) 2020-12-01
AR117861A1 (es) 2021-09-01
EP4523750A2 (en) 2025-03-19

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