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MX2022011334A - Tratamiento de la enfermedad de fabry en pacientes no tratados y tratados previamente con ert. - Google Patents

Tratamiento de la enfermedad de fabry en pacientes no tratados y tratados previamente con ert.

Info

Publication number
MX2022011334A
MX2022011334A MX2022011334A MX2022011334A MX2022011334A MX 2022011334 A MX2022011334 A MX 2022011334A MX 2022011334 A MX2022011334 A MX 2022011334A MX 2022011334 A MX2022011334 A MX 2022011334A MX 2022011334 A MX2022011334 A MX 2022011334A
Authority
MX
Mexico
Prior art keywords
ert
treatment
fabry disease
naã
experienced patients
Prior art date
Application number
MX2022011334A
Other languages
English (en)
Inventor
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
Priority claimed from US15/213,920 external-priority patent/US9999618B2/en
Application filed by Amicus Therapeutics Inc filed Critical Amicus Therapeutics Inc
Publication of MX2022011334A publication Critical patent/MX2022011334A/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
    • 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
    • A61K31/45Non condensed piperidines, e.g. piperocaine having oxo groups directly attached to the heterocyclic ring, e.g. cycloheximide
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7008Compounds having an amino group directly attached to a carbon atom of the saccharide radical, e.g. D-galactosamine, ranimustine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P39/00General protective or antinoxious agents
    • A61P39/02Antidotes
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • General Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Urology & Nephrology (AREA)
  • Toxicology (AREA)
  • Molecular Biology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Nutrition Science (AREA)
  • Physiology (AREA)

Abstract

Se proveen regímenes de dosificación para el tratamiento de la enfermedad de Fabry en un paciente. Algunos métodos se relacionan con el tratamiento de pacientes con Fabry no tratados previamente o tratados con ERT. Algunos métodos comprenden administrar al paciente aproximadamente 123 mg de equivalentes de base libre de migalastat para mejorar la masa ventricular izquierda y/o para mejorar la globotriaosilceramida en podocitos.
MX2022011334A 2016-07-19 2018-06-20 Tratamiento de la enfermedad de fabry en pacientes no tratados y tratados previamente con ert. MX2022011334A (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US15/213,920 US9999618B2 (en) 2007-04-26 2016-07-19 Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones

Publications (1)

Publication Number Publication Date
MX2022011334A true MX2022011334A (es) 2022-10-07

Family

ID=59416847

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2018007669A MX395523B (es) 2016-07-19 2017-07-19 Tratamiento de la enfermedad de fabry en pacientes no tratados y tratados previamente con ert.
MX2022011334A MX2022011334A (es) 2016-07-19 2018-06-20 Tratamiento de la enfermedad de fabry en pacientes no tratados y tratados previamente con ert.

Family Applications Before (1)

Application Number Title Priority Date Filing Date
MX2018007669A MX395523B (es) 2016-07-19 2017-07-19 Tratamiento de la enfermedad de fabry en pacientes no tratados y tratados previamente con ert.

Country Status (15)

Country Link
US (2) US12070453B2 (es)
EP (5) EP4324521A3 (es)
JP (4) JP6752270B2 (es)
KR (3) KR20240017126A (es)
CN (1) CN109661230A (es)
AR (1) AR109103A1 (es)
AU (3) AU2017298376A1 (es)
CA (1) CA3031249A1 (es)
CL (1) CL2018001677A1 (es)
CO (1) CO2018006358A2 (es)
IL (2) IL313907A (es)
MX (2) MX395523B (es)
NZ (2) NZ750752A (es)
TW (2) TWI875676B (es)
WO (1) WO2018017721A1 (es)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2024745B1 (en) 2006-05-16 2015-07-08 Amicus Therapeutics, Inc. Assays for diagnosing and evaluating treatment options for fabry disease
SI2252313T1 (sl) 2008-02-12 2015-08-31 Amicus Therapeutics, Inc. Postopek napovedovanja odgovora na zdravljenje bolezni s farmakološkim spremljevalcem
TWI875676B (zh) * 2016-07-19 2025-03-11 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
US12020820B1 (en) * 2017-03-03 2024-06-25 Cerner Innovation, Inc. Predicting sphingolipidoses (fabry's disease) and decision support
KR20240017111A (ko) 2017-05-30 2024-02-06 아미쿠스 세라퓨틱스, 인코포레이티드 신장 손상을 갖는 파브리 환자를 치료하는 방법
EP4374918B1 (en) 2017-05-30 2025-07-16 Amicus Therapeutics, Inc. Methods of treating fabry patients having renal impairment
WO2019157047A1 (en) * 2018-02-06 2019-08-15 Amicus Therapeutics, Inc. Use of migalastat for treating fabry disease in pregnant patients
LT3749308T (lt) * 2018-02-06 2024-02-12 Amicus Therapeutics, Inc. Klasikine fabri liga sergančių pacientų gydymas migalastatu
SI3840753T1 (sl) * 2018-08-20 2025-01-31 Amicus Therapeutics, Inc. Migalastat za uporabo v postopkih zdravljenja Fabryjeve bolezni pri bolnikih z mutacijo gena GLA
TW202042812A (zh) * 2019-01-22 2020-12-01 美商阿米庫斯醫療股份有限公司 減少法布瑞氏症患者中之腦血管事件之方法
MX2021015352A (es) * 2019-06-11 2022-04-06 Amicus Therapeutics Inc Métodos para tratar la enfermedad de fabry en pacientes que tienen insuficiencia renal.
BR112022002202A2 (pt) 2019-08-07 2022-06-14 Amicus Therapeutics Inc Métodos de tratar doença de fabry em pacientes tendo uma mutação no gene gla
US20210251972A1 (en) * 2020-02-10 2021-08-19 Amicus Therapeutics, Inc. Methods Of Treating Fabry Disease
US11623916B2 (en) 2020-12-16 2023-04-11 Amicus Therapeutics, Inc. Highly purified batches of pharmaceutical grade migalastat and methods of producing the same
KR102471225B1 (ko) 2021-11-18 2022-11-28 박기용 어플리케이션을 통한 소비자 및 농장 연결 시스템

Family Cites Families (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0463109A4 (en) 1989-03-24 1992-11-19 Research Corporation Technologies, Inc. Recombinant alpha-galactosidase, a therapy for fabry disease
EP0536402A4 (en) 1990-06-29 1993-05-12 Nippon Shinyaku Company, Limited Piperidine derivative
US6270954B1 (en) 1996-04-10 2001-08-07 The Regents Of The University Of California Correction of genetic defects using chemical chaperones
JP2000509971A (ja) 1996-04-10 2000-08-08 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 化学的シャペローンを用いる遺伝的欠陥の修正
US6274597B1 (en) 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
WO2004069190A2 (en) 2003-01-31 2004-08-19 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiency disorders
US7446098B2 (en) 2003-02-18 2008-11-04 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiencies
BRPI0416216A (pt) 2003-11-12 2006-12-26 Amicus Therapeutics Inc composto de glicoimidazol e polihidroxiciclohexenila e método para sintetizar composto de glicoimidazol e polihidroxiciclohexenila, métodos para inibir glicocerebrosidase, para aumentar a atividade de glicocerebrosidase e para estabilizar glicocerebrosidade, composição farmacêutica, métodos para inibir uma -glicosidade, e para tratar doença de gaucher, derivado de glicoimidazol e derivados de polihidroxiciclohexilamina
EP3441090A1 (en) 2005-05-17 2019-02-13 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
MX2007015602A (es) 2005-06-08 2008-02-21 Amicus Therapeutics Inc Tratamiento de trastornos del sistema nervioso central asociados con mutaciones en genes que codifican las enzimas lisosomicas.
US7955262B2 (en) 2005-07-26 2011-06-07 Syneron Medical Ltd. Method and apparatus for treatment of skin using RF and ultrasound energies
AU2006272497B2 (en) 2005-07-27 2012-07-19 University Of Florida Research Foundation, Inc. Small compounds that correct protein misfolding and uses thereof
AU2015230773B2 (en) 2006-05-16 2017-09-28 Amicus Therapeutics Inc. Assays for diagnosing and evaluating treatment options for fabry disease
EP2024745B1 (en) * 2006-05-16 2015-07-08 Amicus Therapeutics, Inc. Assays for diagnosing and evaluating treatment options for fabry disease
WO2008034628A1 (en) 2006-09-22 2008-03-27 Krka, Tovarna Zdravil, D.D., Novo Mesto Aripiprazole hemifumarate and process for its preparation
JP2010523578A (ja) 2007-03-30 2010-07-15 アミカス セラピューティックス インコーポレイテッド 薬理シャペロンを用いるファブリー病の治療方法
SI2252313T1 (sl) 2008-02-12 2015-08-31 Amicus Therapeutics, Inc. Postopek napovedovanja odgovora na zdravljenje bolezni s farmakološkim spremljevalcem
EA031874B1 (ru) * 2011-03-11 2019-03-29 Амикус Терапьютикс, Инк. Способ лечения болезни фабри
JP2013255488A (ja) * 2012-05-18 2013-12-26 Saitama Medical Univ 拡張期心不全非ヒトモデル動物及びその製造方法、並びに、拡張期心不全予防乃至症状改善活性を有する物質のスクリーニング方法
TWI875676B (zh) * 2016-07-19 2025-03-11 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

Also Published As

Publication number Publication date
JP2019178140A (ja) 2019-10-17
TW202408513A (zh) 2024-03-01
NZ750752A (en) 2025-07-25
US20190183869A1 (en) 2019-06-20
EP4356911A1 (en) 2024-04-24
TWI875676B (zh) 2025-03-11
EP4324521A2 (en) 2024-02-21
KR20230061563A (ko) 2023-05-08
JP2024026079A (ja) 2024-02-28
AU2023206175B2 (en) 2025-05-22
NZ790449A (en) 2025-08-29
MX395523B (es) 2025-03-25
IL313907A (en) 2024-08-01
EP4324521A3 (en) 2024-04-24
MX2018007669A (es) 2018-09-12
AU2017298376A1 (en) 2019-03-07
KR20240017126A (ko) 2024-02-06
US20230321065A1 (en) 2023-10-12
KR20190030729A (ko) 2019-03-22
CO2018006358A2 (es) 2018-11-30
US12070453B2 (en) 2024-08-27
EP4389213A3 (en) 2024-08-21
WO2018017721A1 (en) 2018-01-25
EP4378524A2 (en) 2024-06-05
JP2018526368A (ja) 2018-09-13
JP6752270B2 (ja) 2020-09-09
EP3487502A1 (en) 2019-05-29
AU2023206175A1 (en) 2023-08-10
EP4389213A2 (en) 2024-06-26
TW201806597A (zh) 2018-03-01
CA3031249A1 (en) 2018-01-25
JP2022033749A (ja) 2022-03-02
CN109661230A (zh) 2019-04-19
AR109103A1 (es) 2018-10-31
CL2018001677A1 (es) 2019-02-01
EP4378524A3 (en) 2024-08-14
IL264301A (en) 2019-02-28
AU2025217396A1 (en) 2025-09-04

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