AR112482A1 - Métodos para aumentar y/o estabilizar la función cardiaca en pacientes con enfermedad de fabry - Google Patents

Métodos para aumentar y/o estabilizar la función cardiaca en pacientes con enfermedad de fabry

Info

Publication number
AR112482A1
AR112482A1 ARP180102438A AR112482A1 AR 112482 A1 AR112482 A1 AR 112482A1 AR P180102438 A ARP180102438 A AR P180102438A AR 112482 A1 AR112482 A1 AR 112482A1
Authority
AR
Argentina
Prior art keywords
cardiac function
fabry
disease
patients
methods
Prior art date
Application number
Other languages
English (en)
Inventor
Nina Skuban
Jay Barth
Jeff Castelli
Original Assignee
Amicus Therapeutics Inc
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 Amicus Therapeutics Inc filed Critical Amicus Therapeutics Inc
Publication of AR112482A1 publication Critical patent/AR112482A1/es

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

Abstract

Reivindicación 1: Un método para mejorar la función cardiaca en un paciente que tiene enfermedad de Fabry, en donde el método comprende administrar al paciente una formulación que comprende una cantidad eficaz de migalastat o una sal del mismo cada dos días para mejorar la función cardiaca del paciente, en donde la cantidad eficaz es de entre aproximadamente 100 mg y aproximadamente 150 mg equivalentes de base libre (FBE).
ARP180102438 2017-08-28 2018-08-28 Métodos para aumentar y/o estabilizar la función cardiaca en pacientes con enfermedad de fabry AR112482A1 (es)

Applications Claiming Priority (1)

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

Publications (1)

Publication Number Publication Date
AR112482A1 true AR112482A1 (es) 2019-10-30

Family

ID=63528948

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP180102438 AR112482A1 (es) 2017-08-28 2018-08-28 Métodos para aumentar y/o estabilizar la función cardiaca en pacientes con enfermedad de fabry

Country Status (23)

Country Link
US (1) US20200222377A1 (es)
EP (2) EP3675853B1 (es)
JP (2) JP2020531550A (es)
KR (1) KR20200044908A (es)
CN (1) CN111770753A (es)
AR (1) AR112482A1 (es)
AU (1) AU2018326364A1 (es)
BR (1) BR112020003974A2 (es)
CA (1) CA3074450A1 (es)
CL (1) CL2020000491A1 (es)
DK (1) DK3675853T3 (es)
EA (1) EA202090564A1 (es)
ES (1) ES2931054T3 (es)
HR (1) HRP20221366T1 (es)
HU (1) HUE060287T2 (es)
IL (2) IL310648A (es)
LT (1) LT3675853T (es)
MX (2) MX2020002229A (es)
PL (1) PL3675853T3 (es)
PT (1) PT3675853T (es)
RS (1) RS63742B1 (es)
SG (1) SG11202001707PA (es)
WO (1) WO2019046244A1 (es)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SI2533050T1 (sl) 2006-05-16 2014-07-31 Amicus Therapeutics, Inc. MoĹľnosti zdravljenja Fabrijeve bolezni
PT2252313E (pt) 2008-02-12 2015-08-26 Amicus Therapeutics Inc Método para previsão da resposta ao tratamento farmacológico de doenças com chaperonas
MX2019014410A (es) 2017-05-30 2020-02-10 Amicus Therapeutics Inc Metodos de tratamiento de pacientes con enfermedad de fabry que tienen insuficiencia renal.
KR20200128676A (ko) 2018-02-06 2020-11-16 아미쿠스 세라퓨틱스, 인코포레이티드 임신 환자의 파브리병 치료를 위한 미갈라스타트의 용도
AU2020327019A1 (en) 2019-08-07 2022-03-03 Amicus Therapeutics, Inc. Methods of treating Fabry disease in patients having a mutation in the GLA gene
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
EP2142197A4 (en) 2007-03-30 2010-11-10 Amicus Therapeutics Inc PROCESS FOR THE TREATMENT OF FABRY DISEASE USING PHARMACOLOGICAL CHAPERONS
US9999618B2 (en) * 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
PT2252313E (pt) 2008-02-12 2015-08-26 Amicus Therapeutics Inc Método para previsão da resposta ao tratamento farmacológico de doenças com chaperonas

Also Published As

Publication number Publication date
ES2931054T3 (es) 2022-12-23
PT3675853T (pt) 2022-11-28
EP4176877A1 (en) 2023-05-10
TW201919619A (zh) 2019-06-01
WO2019046244A1 (en) 2019-03-07
EA202090564A1 (ru) 2020-06-15
MX2020002229A (es) 2020-07-20
SG11202001707PA (en) 2020-03-30
CA3074450A1 (en) 2019-03-07
EP3675853A1 (en) 2020-07-08
JP2020531550A (ja) 2020-11-05
DK3675853T3 (da) 2022-11-28
KR20200044908A (ko) 2020-04-29
IL310648A (en) 2024-04-01
CN111770753A (zh) 2020-10-13
HUE060287T2 (hu) 2023-02-28
PL3675853T3 (pl) 2022-12-27
JP2023051998A (ja) 2023-04-11
IL272893B1 (en) 2024-03-01
EP3675853B1 (en) 2022-09-07
MX2023005628A (es) 2023-05-24
HRP20221366T1 (hr) 2023-01-06
RS63742B1 (sr) 2022-12-30
IL272893A (en) 2020-04-30
BR112020003974A2 (pt) 2020-09-01
LT3675853T (lt) 2022-12-12
CL2020000491A1 (es) 2020-09-11
AU2018326364A1 (en) 2020-04-09
US20200222377A1 (en) 2020-07-16

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