JP7755929B2 - ファブリー病患者における心臓機能の強化及び／または安定化方法 - Google Patents

ファブリー病患者における心臓機能の強化及び／または安定化方法

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JP7755929B2
JP7755929B2 JP2020511971A JP2020511971A JP7755929B2 JP 7755929 B2 JP7755929 B2 JP 7755929B2 JP 2020511971 A JP2020511971 A JP 2020511971A JP 2020511971 A JP2020511971 A JP 2020511971A JP 7755929 B2 JP7755929 B2 JP 7755929B2
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migalastat
patient
salt
mwfs
patients
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JP2020531550A (ja
JP2020531550A5 (https=
Inventor
ジェフ カステリ，
ジェイ バース，
ニーナ スクバン，
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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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Priority to JP2022203783A priority Critical patent/JP2023051998A/ja
Priority to JP2025088523A priority patent/JP2025138638A/ja
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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
    • 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
    • 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
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/08—Solutions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/20—Pills, tablets, discs, rods
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate

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  • Health & Medical Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Cardiology (AREA)
  • Epidemiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Hospice & Palliative Care (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2020511971A 2017-08-28 2018-08-28 ファブリー病患者における心臓機能の強化及び／または安定化方法 Active JP7755929B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2022203783A JP2023051998A (ja) 2017-08-28 2022-12-20 ファブリー病患者における心臓機能の強化及び／または安定化方法
JP2025088523A JP2025138638A (ja) 2017-08-28 2025-05-28 ファブリー病患者における心臓機能の強化及び／または安定化方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762550984P 2017-08-28 2017-08-28
US62/550,984 2017-08-28
PCT/US2018/048257 WO2019046244A1 (en) 2017-08-28 2018-08-28 METHODS FOR ENHANCING AND / OR STABILIZING CARDIAC FUNCTION IN PATIENTS WITH FABRY DISEASE

Related Child Applications (2)

Application Number Title Priority Date Filing Date
JP2022203783A Division JP2023051998A (ja) 2017-08-28 2022-12-20 ファブリー病患者における心臓機能の強化及び／または安定化方法
JP2025088523A Division JP2025138638A (ja) 2017-08-28 2025-05-28 ファブリー病患者における心臓機能の強化及び／または安定化方法

Publications (3)

Publication Number Publication Date
JP2020531550A JP2020531550A (ja) 2020-11-05
JP2020531550A5 JP2020531550A5 (https=) 2025-06-10
JP7755929B2 true JP7755929B2 (ja) 2025-10-17

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ID=63528948

Family Applications (3)

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JP2020511971A Active JP7755929B2 (ja) 2017-08-28 2018-08-28 ファブリー病患者における心臓機能の強化及び／または安定化方法
JP2022203783A Pending JP2023051998A (ja) 2017-08-28 2022-12-20 ファブリー病患者における心臓機能の強化及び／または安定化方法
JP2025088523A Pending JP2025138638A (ja) 2017-08-28 2025-05-28 ファブリー病患者における心臓機能の強化及び／または安定化方法

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2022203783A Pending JP2023051998A (ja) 2017-08-28 2022-12-20 ファブリー病患者における心臓機能の強化及び／または安定化方法
JP2025088523A Pending JP2025138638A (ja) 2017-08-28 2025-05-28 ファブリー病患者における心臓機能の強化及び／または安定化方法

Country Status (26)

Country Link
US (1) US20200222377A1 (https=)
EP (3) EP3675853B1 (https=)
JP (3) JP7755929B2 (https=)
KR (2) KR20250049561A (https=)
CN (1) CN111770753A (https=)
AR (1) AR112482A1 (https=)
AU (2) AU2018326364B2 (https=)
CA (1) CA3074450C (https=)
CL (1) CL2020000491A1 (https=)
DK (2) DK3675853T3 (https=)
EA (1) EA202090564A1 (https=)
ES (2) ES2931054T3 (https=)
FI (1) FI4176877T3 (https=)
HR (2) HRP20250064T1 (https=)
HU (2) HUE060287T2 (https=)
IL (3) IL272893B2 (https=)
LT (2) LT3675853T (https=)
MX (2) MX2020002229A (https=)
PL (2) PL4176877T3 (https=)
PT (2) PT3675853T (https=)
RS (2) RS66515B1 (https=)
SG (1) SG11202001707PA (https=)
SI (1) SI4176877T1 (https=)
SM (2) SMT202500073T1 (https=)
TW (1) TWI845479B (https=)
WO (1) WO2019046244A1 (https=)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL2533050T6 (pl) 2006-05-16 2016-05-31 Amicus Therapeutics Inc Możliwości leczenia choroby Fabry'ego
EP2946785B1 (en) 2008-02-12 2018-10-24 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
CA3224546C (en) 2017-05-30 2026-02-24 Amicus Therapeutics, Inc. Use of migalastat in the treatment of fabry patients
US20200222377A1 (en) 2017-08-28 2020-07-16 Amicus Therapeutics, Inc. Methods Of Enhancing And/Or Stabilizing Cardiac Function In Patients With Fabry Disease
KR20200128676A (ko) 2018-02-06 2020-11-16 아미쿠스 세라퓨틱스, 인코포레이티드 임신 환자의 파브리병 치료를 위한 미갈라스타트의 용도
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
TR2024011149A1 (tr) 2024-08-23 2026-03-23 Sanovel İlaç Sanayi̇ Ve Ti̇caret Anoni̇m Şi̇rketi̇ Mi̇galastat i̇çeren kapsül formülasyonlari
CN119120696A (zh) * 2024-11-12 2024-12-13 北京贝瑞和康生物技术有限公司 检测法布雷病gla基因突变的引物组、试剂盒及其应用

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160324839A1 (en) 2007-04-26 2016-11-10 Amicus Therapeutics, Inc. Dosing Regimens for the Treatment of Lysosomal Storage Diseases Using Pharmacological Chaperones

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.
EP2946785B1 (en) 2008-02-12 2018-10-24 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
US20200222377A1 (en) 2017-08-28 2020-07-16 Amicus Therapeutics, Inc. Methods Of Enhancing And/Or Stabilizing Cardiac Function In Patients With Fabry Disease
EP3676914B1 (en) 2017-08-28 2023-08-16 John Mezzalingua Associates LLC Weather protecting (wp) housing for coaxial cable connectors

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160324839A1 (en) 2007-04-26 2016-11-10 Amicus Therapeutics, Inc. Dosing Regimens for the Treatment of Lysosomal Storage Diseases Using Pharmacological Chaperones

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
Drug Design, Development and Therapy, 2016年, 発行日, Vol.10, pp.1771-1781
J Med Genet, 2017年, 発行日, Vol.54, pp.288-298
JIMD Reports, 2015年, 発行日, Vol.23, pp.7-15
Lancet, 2009年, 発行日, Vol.374, pp.1986-1996
N Engl J Med, 2016年, 発行日, Vol.375, pp.545-555

Also Published As

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US20200222377A1 (en) 2020-07-16
MX2023005628A (es) 2023-05-24
SMT202500073T1 (it) 2025-03-12
CA3074450C (en) 2024-10-01
JP2020531550A (ja) 2020-11-05
AR112482A1 (es) 2019-10-30
SG11202001707PA (en) 2020-03-30
RS63742B1 (sr) 2022-12-30
ES3013820T3 (en) 2025-04-15
AU2018326364B2 (en) 2024-08-29
IL272893A (en) 2020-04-30
CN111770753A (zh) 2020-10-13
EP4176877A1 (en) 2023-05-10
AU2018326364A1 (en) 2020-04-09
IL272893B2 (en) 2024-07-01
DK3675853T3 (da) 2022-11-28
FI4176877T3 (fi) 2025-03-06
LT4176877T (lt) 2025-02-10
HUE070433T2 (hu) 2025-06-28
EP4176877B1 (en) 2024-12-04
MX2020002229A (es) 2020-07-20
IL310648A (en) 2024-04-01
LT3675853T (lt) 2022-12-12
KR20200044908A (ko) 2020-04-29
TWI845479B (zh) 2024-06-21
BR112020003974A2 (pt) 2020-09-01
CL2020000491A1 (es) 2020-09-11
KR102790184B1 (ko) 2025-04-01
CA3074450A1 (en) 2019-03-07
PT3675853T (pt) 2022-11-28
WO2019046244A1 (en) 2019-03-07
HUE060287T2 (hu) 2023-02-28
AU2024266945A1 (en) 2024-12-19
JP2025138638A (ja) 2025-09-25
HRP20221366T1 (hr) 2023-01-06
IL272893B1 (en) 2024-03-01
JP2023051998A (ja) 2023-04-11
EP3675853A1 (en) 2020-07-08
HRP20250064T1 (hr) 2025-03-14
SI4176877T1 (sl) 2025-03-31
EA202090564A1 (ru) 2020-06-15
KR20250049561A (ko) 2025-04-11
RS66515B1 (sr) 2025-03-31
PL3675853T3 (pl) 2022-12-27
EP4588476A1 (en) 2025-07-23
PT4176877T (pt) 2025-02-24
IL324182A (en) 2025-12-01
EP3675853B1 (en) 2022-09-07
SMT202200459T1 (it) 2023-01-13
TW201919619A (zh) 2019-06-01
PL4176877T3 (pl) 2025-04-07
DK4176877T3 (da) 2025-02-24
ES2931054T3 (es) 2022-12-23

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