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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Publication number
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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Japanese (ja)
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JP2020531550A (ja
JP2020531550A5 (enExample
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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Application filed by アミカス セラピューティックス インコーポレイテッド filed Critical アミカス セラピューティックス インコーポレイテッド
Publication of JP2020531550A publication Critical patent/JP2020531550A/ja
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)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (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 (enExample) 2025-06-10
JP7755929B2 true JP7755929B2 (ja) 2025-10-17

Family

ID=63528948

Family Applications (3)

Application Number Title Priority Date Filing Date
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 (enExample)
EP (3) EP3675853B1 (enExample)
JP (3) JP7755929B2 (enExample)
KR (2) KR102790184B1 (enExample)
CN (1) CN111770753A (enExample)
AR (1) AR112482A1 (enExample)
AU (2) AU2018326364B2 (enExample)
CA (1) CA3074450C (enExample)
CL (1) CL2020000491A1 (enExample)
DK (2) DK4176877T3 (enExample)
EA (1) EA202090564A1 (enExample)
ES (2) ES2931054T3 (enExample)
FI (1) FI4176877T3 (enExample)
HR (2) HRP20250064T1 (enExample)
HU (2) HUE070433T2 (enExample)
IL (3) IL272893B2 (enExample)
LT (2) LT4176877T (enExample)
MX (2) MX2020002229A (enExample)
PL (2) PL4176877T3 (enExample)
PT (2) PT4176877T (enExample)
RS (2) RS66515B1 (enExample)
SG (1) SG11202001707PA (enExample)
SI (1) SI4176877T1 (enExample)
SM (2) SMT202500073T1 (enExample)
TW (1) TWI845479B (enExample)
WO (1) WO2019046244A1 (enExample)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX351004B (es) 2006-05-16 2017-09-26 Nat Institutes Of Health Ensayos para diagnosticar y evaluar opciones de tratamiento para enfermedad de fabry.
WO2009102895A2 (en) 2008-02-12 2009-08-20 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
AU2018277756A1 (en) 2017-05-30 2020-01-23 Amicus Therapeutics, Inc. Methods of treating fabry patients having renal impairment
WO2019046244A1 (en) 2017-08-28 2019-03-07 Amicus Therapeutics, Inc. METHODS FOR ENHANCING AND / OR STABILIZING CARDIAC FUNCTION IN PATIENTS WITH FABRY DISEASE
EP4717319A3 (en) 2018-02-06 2026-06-10 Amicus Therapeutics, Inc. Methods of treating fabry disease in pregnant patients
LT3840753T (lt) 2018-08-20 2024-11-11 Amicus Therapeutics, Inc. Migalastatas skirtas naudoti fabry ligos gydymo būdui, pacientams turintiems gla geno mutaciją
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 Ilac Sanayi Ve Ticaret Anonim Sirketi 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
EP2142197A4 (en) 2007-03-30 2010-11-10 Amicus Therapeutics Inc METHOD FOR THE TREATMENT OF MORBUS FABRY WITH PHARMACOLOGICAL CHAPTERONES
WO2009102895A2 (en) 2008-02-12 2009-08-20 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
WO2019046244A1 (en) 2017-08-28 2019-03-07 Amicus Therapeutics, Inc. METHODS FOR ENHANCING AND / OR STABILIZING CARDIAC FUNCTION IN PATIENTS WITH FABRY DISEASE
US10811814B2 (en) 2017-08-28 2020-10-20 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

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

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