EA202090564A1 - Способы усиления и/или стабилизации сердечной функции у пациентов с болезнью фабри - Google Patents

Способы усиления и/или стабилизации сердечной функции у пациентов с болезнью фабри

Info

Publication number
EA202090564A1
EA202090564A1 EA202090564A EA202090564A EA202090564A1 EA 202090564 A1 EA202090564 A1 EA 202090564A1 EA 202090564 A EA202090564 A EA 202090564A EA 202090564 A EA202090564 A EA 202090564A EA 202090564 A1 EA202090564 A1 EA 202090564A1
Authority
EA
Eurasian Patent Office
Prior art keywords
methods
cardiac function
disease
patients
strengthening
Prior art date
Application number
EA202090564A
Other languages
English (en)
Inventor
Джефф Кастелли
Джей Барт
Нина Скубан
Original Assignee
Амикус Терапьютикс, Инк.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Амикус Терапьютикс, Инк. filed Critical Амикус Терапьютикс, Инк.
Publication of EA202090564A1 publication Critical patent/EA202090564A1/ru

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic 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
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445Non condensed piperidines, e.g. piperocaine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/04Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/08Solutions
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/20Pills, tablets, discs, rods
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate

Landscapes

  • Health & Medical Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)

Abstract

Предоставлены способы лечения болезни Фабри у пациента. Определенные способы относятся к лечению ФЗТ (ферментная заместительная терапия)-леченных или ФЗТ-наивных пациентов с болезнью Фабри. Определенные способы включают введение пациенту от около 100 до около 150 мг эквивалента свободного основания мигаластата для усиления и/или стабилизации сердечной функции.
EA202090564A 2017-08-28 2018-08-28 Способы усиления и/или стабилизации сердечной функции у пациентов с болезнью фабри EA202090564A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762550984P 2017-08-28 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

Publications (1)

Publication Number Publication Date
EA202090564A1 true EA202090564A1 (ru) 2020-06-15

Family

ID=63528948

Family Applications (1)

Application Number Title Priority Date Filing Date
EA202090564A EA202090564A1 (ru) 2017-08-28 2018-08-28 Способы усиления и/или стабилизации сердечной функции у пациентов с болезнью фабри

Country Status (23)

Country Link
US (1) US20200222377A1 (ru)
EP (2) EP4176877A1 (ru)
JP (2) JP2020531550A (ru)
KR (1) KR20200044908A (ru)
CN (1) CN111770753A (ru)
AR (1) AR112482A1 (ru)
AU (1) AU2018326364A1 (ru)
BR (1) BR112020003974A2 (ru)
CA (1) CA3074450A1 (ru)
CL (1) CL2020000491A1 (ru)
DK (1) DK3675853T3 (ru)
EA (1) EA202090564A1 (ru)
ES (1) ES2931054T3 (ru)
HR (1) HRP20221366T1 (ru)
HU (1) HUE060287T2 (ru)
IL (2) IL310648A (ru)
LT (1) LT3675853T (ru)
MX (2) MX2020002229A (ru)
PL (1) PL3675853T3 (ru)
PT (1) PT3675853T (ru)
RS (1) RS63742B1 (ru)
SG (1) SG11202001707PA (ru)
WO (1) WO2019046244A1 (ru)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL2787345T3 (pl) 2006-05-16 2016-08-31 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
WO2018222655A1 (en) 2017-05-30 2018-12-06 Amicus Therapeutics, Inc. Methods of treating fabry patients having renal impairment
EP3749307A1 (en) 2018-02-06 2020-12-16 Amicus Therapeutics, Inc. Use of migalastat for treating fabry disease in pregnant patients
TW202120087A (zh) 2019-08-07 2021-06-01 美商阿米庫斯醫療股份有限公司 治療在GLA基因中具有突變的患者的Fabry氏病之方法
US11623916B2 (en) 2020-12-16 2023-04-11 Amicus Therapeutics, Inc. Highly purified batches of pharmaceutical grade migalastat and methods of producing the same

Family Cites Families (4)

* 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
US20100113517A1 (en) 2007-03-30 2010-05-06 Amicus Therapeutics, Inc. Method for the treatment of fabry disease using pharmacological chaperones
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

Also Published As

Publication number Publication date
CL2020000491A1 (es) 2020-09-11
MX2020002229A (es) 2020-07-20
ES2931054T3 (es) 2022-12-23
BR112020003974A2 (pt) 2020-09-01
CA3074450A1 (en) 2019-03-07
WO2019046244A1 (en) 2019-03-07
PT3675853T (pt) 2022-11-28
HRP20221366T1 (hr) 2023-01-06
SG11202001707PA (en) 2020-03-30
MX2023005628A (es) 2023-05-24
EP3675853A1 (en) 2020-07-08
EP3675853B1 (en) 2022-09-07
AU2018326364A1 (en) 2020-04-09
RS63742B1 (sr) 2022-12-30
IL272893B1 (en) 2024-03-01
JP2020531550A (ja) 2020-11-05
EP4176877A1 (en) 2023-05-10
TW201919619A (zh) 2019-06-01
US20200222377A1 (en) 2020-07-16
CN111770753A (zh) 2020-10-13
LT3675853T (lt) 2022-12-12
JP2023051998A (ja) 2023-04-11
HUE060287T2 (hu) 2023-02-28
AR112482A1 (es) 2019-10-30
IL272893A (en) 2020-04-30
KR20200044908A (ko) 2020-04-29
DK3675853T3 (da) 2022-11-28
IL310648A (en) 2024-04-01
PL3675853T3 (pl) 2022-12-27
IL272893B2 (en) 2024-07-01

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