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CL2018001677A1 - 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
CL2018001677A1
CL2018001677A1 CL2018001677A CL2018001677A CL2018001677A1 CL 2018001677 A1 CL2018001677 A1 CL 2018001677A1 CL 2018001677 A CL2018001677 A CL 2018001677A CL 2018001677 A CL2018001677 A CL 2018001677A CL 2018001677 A1 CL2018001677 A1 CL 2018001677A1
Authority
CL
Chile
Prior art keywords
treated
treatment
ert
patients
fabry disease
Prior art date
Application number
CL2018001677A
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 CL2018001677A1 publication Critical patent/CL2018001677A1/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
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Urology & Nephrology (AREA)
  • Cardiology (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.
CL2018001677A 2016-07-19 2018-06-20 Tratamiento de la enfermedad de fabry en pacientes no tratados y tratados previamente con ert CL2018001677A1 (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
CL2018001677A1 true CL2018001677A1 (es) 2019-02-01

Family

ID=59416847

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2018001677A CL2018001677A1 (es) 2016-07-19 2018-06-20 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) EP4356911A1 (es)
JP (4) JP6752270B2 (es)
KR (3) KR20230061563A (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
HUE027569T2 (en) 2006-05-16 2016-10-28 Amicus Therapeutics Inc Preparation for the treatment of Fabry disease
CA2715407C (en) 2008-02-12 2022-07-26 Elfrida Benjamin Method to predict response to pharmacological chaperone treatment of diseases
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
AU2018277756A1 (en) 2017-05-30 2020-01-23 Amicus Therapeutics, Inc. Methods of treating fabry patients having renal impairment
SI4062916T1 (sl) 2017-05-30 2025-04-30 Amicus Therapeutics, Inc. Zdravljenje bolnikov s Fabryjem, ki imajo določene mutacije
EP3749307A1 (en) * 2018-02-06 2020-12-16 Amicus Therapeutics, Inc. Use of migalastat for treating fabry disease in pregnant patients
CA3090499A1 (en) * 2018-02-06 2019-08-15 Amicus Therapeutics, Inc. Use of migalastat for the treatment of patients with classic fabry disease
MX2021002070A (es) * 2018-08-20 2021-07-15 Amicus Therapeutics Inc Metodos para tratar la enfemedad de fabry en pacientes que tienen una mutacion en el gen 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.
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
EP4103178A4 (en) * 2020-02-10 2024-03-20 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
WO1990011353A1 (en) 1989-03-24 1990-10-04 Research Corporation Technologies, Inc. Recombinant alpha-galactosidase, a therapy for fabry disease
WO1992000277A1 (en) 1990-06-29 1992-01-09 Nippon Shinyaku Co., Ltd. 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
MX344587B (es) 2003-01-31 2016-12-20 Mount Sinai School Of Medicine Of New York Univ Metodo para incrementar la estabilidad in vitro e in vivo de enzimas asociadas al transtorno lisosomal.
US7446098B2 (en) 2003-02-18 2008-11-04 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiencies
CA2545435C (en) 2003-11-12 2014-08-19 Amicus Therapeutics Inc. Hydroxy piperidine derivatives to treat gaucher disease
ES2572148T3 (es) 2005-05-17 2016-05-30 Amicus Therapeutics Inc Un método para el tratamiento de la enfermedad de Pompe usando 1-desoxinojirimicina y derivados
CN104771402A (zh) 2005-06-08 2015-07-15 阿米库斯治疗学公司 溶酶体酶编码基因突变相关的cns紊乱的治疗
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
HUE027569T2 (en) * 2006-05-16 2016-10-28 Amicus Therapeutics Inc Preparation for the treatment of Fabry disease
AU2015230773B2 (en) 2006-05-16 2017-09-28 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
AU2008232614A1 (en) 2007-03-30 2008-10-09 Amicus Therapeutics, Inc. Method for the treatment of Fabry disease using pharmacological chaperones
CA2715407C (en) 2008-02-12 2022-07-26 Elfrida Benjamin Method to predict response to pharmacological chaperone treatment of diseases
CA2829947C (en) * 2011-03-11 2019-01-15 Amicus Therapeutics, Inc. Dosing regimens for the treatment of fabry disease
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
IL264301A (en) 2019-02-28
AU2025217396A1 (en) 2025-09-04
NZ750752A (en) 2025-07-25
TW202408513A (zh) 2024-03-01
IL313907A (en) 2024-08-01
EP3487502A1 (en) 2019-05-29
KR20190030729A (ko) 2019-03-22
JP2022033749A (ja) 2022-03-02
JP6752270B2 (ja) 2020-09-09
CO2018006358A2 (es) 2018-11-30
TWI875676B (zh) 2025-03-11
JP2018526368A (ja) 2018-09-13
EP4389213A3 (en) 2024-08-21
AU2023206175B2 (en) 2025-05-22
JP2024026079A (ja) 2024-02-28
MX395523B (es) 2025-03-25
NZ790449A (en) 2025-08-29
AU2017298376A1 (en) 2019-03-07
EP4324521A3 (en) 2024-04-24
MX2022011334A (es) 2022-10-07
EP4324521A2 (en) 2024-02-21
US20230321065A1 (en) 2023-10-12
MX2018007669A (es) 2018-09-12
KR20230061563A (ko) 2023-05-08
US20190183869A1 (en) 2019-06-20
EP4378524A2 (en) 2024-06-05
JP2019178140A (ja) 2019-10-17
EP4378524A3 (en) 2024-08-14
KR20240017126A (ko) 2024-02-06
CA3031249A1 (en) 2018-01-25
US12070453B2 (en) 2024-08-27
TW201806597A (zh) 2018-03-01
CN109661230A (zh) 2019-04-19
AU2023206175A1 (en) 2023-08-10
EP4356911A1 (en) 2024-04-24
AR109103A1 (es) 2018-10-31
EP4389213A2 (en) 2024-06-26
WO2018017721A1 (en) 2018-01-25

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