SG11202101117QA - Methods of treating fabry disease in patients having a mutation in the gla gene - Google Patents

Methods of treating fabry disease in patients having a mutation in the gla gene

Info

Publication number
SG11202101117QA
SG11202101117QA SG11202101117QA SG11202101117QA SG11202101117QA SG 11202101117Q A SG11202101117Q A SG 11202101117QA SG 11202101117Q A SG11202101117Q A SG 11202101117QA SG 11202101117Q A SG11202101117Q A SG 11202101117QA SG 11202101117Q A SG11202101117Q A SG 11202101117QA
Authority
SG
Singapore
Prior art keywords
mutation
patients
methods
fabry disease
treating fabry
Prior art date
Application number
SG11202101117QA
Other languages
English (en)
Inventor
Elfrida Benjamin
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 SG11202101117QA publication Critical patent/SG11202101117QA/en

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
    • 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
    • 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)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Urology & Nephrology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Analytical Chemistry (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Pathology (AREA)
  • Biochemistry (AREA)
SG11202101117QA 2018-08-20 2019-01-16 Methods of treating fabry disease in patients having a mutation in the gla gene SG11202101117QA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862719962P 2018-08-20 2018-08-20
PCT/US2019/013761 WO2020040806A1 (fr) 2018-08-20 2019-01-16 Méthodes de traitement de la maladie de fabry chez les patients porteurs d'une mutation du gène gla

Publications (1)

Publication Number Publication Date
SG11202101117QA true SG11202101117QA (en) 2021-03-30

Family

ID=65279726

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202101117QA SG11202101117QA (en) 2018-08-20 2019-01-16 Methods of treating fabry disease in patients having a mutation in the gla gene

Country Status (14)

Country Link
US (2) US20210315875A1 (fr)
EP (7) EP4282472A3 (fr)
JP (2) JP2021535107A (fr)
KR (3) KR20210046729A (fr)
CN (1) CN112888439A (fr)
AU (1) AU2019326252A1 (fr)
BR (1) BR112021003137A2 (fr)
CA (1) CA3110065A1 (fr)
CL (1) CL2021000435A1 (fr)
EA (1) EA202190569A1 (fr)
IL (1) IL280961A (fr)
MX (2) MX2021002070A (fr)
SG (1) SG11202101117QA (fr)
WO (1) WO2020040806A1 (fr)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2709445T3 (es) 2006-05-16 2019-04-16 Amicus Therapeutics Inc Opciones de tratamiento para la enfermedad de Fabry
PT3470077T (pt) 2008-02-12 2020-11-30 Amicus Therapeutics Inc Método para previsão da resposta ao tratamento farmacológico de doenças com chaperonas
AU2018277756A1 (en) 2017-05-30 2020-01-23 Amicus Therapeutics, Inc. Methods of treating fabry patients having renal impairment
EP3749307A1 (fr) 2018-02-06 2020-12-16 Amicus Therapeutics, Inc. Utilisation de migalastat pour le traitement de la maladie de fabry chez des patientes enceintes
CN114423428A (zh) 2019-08-07 2022-04-29 阿米库斯治疗学公司 治疗在gla基因中具有突变的患者的法布里病的方法
US11623916B2 (en) 2020-12-16 2023-04-11 Amicus Therapeutics, Inc. Highly purified batches of pharmaceutical grade migalastat and methods of producing the same
CN113969313A (zh) * 2021-11-29 2022-01-25 百世诺(北京)医学检验实验室有限公司 罕见遗传病突变基因及其应用
CN116656704A (zh) * 2023-06-01 2023-08-29 华东理工大学 用于治疗法布里病的转基因表达盒
CN116622750B (zh) * 2023-06-01 2024-05-31 上海勉亦生物科技有限公司 优化的人法布里转基因表达盒及其用途

Family Cites Families (12)

* 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 (fr) 2007-03-30 2010-11-10 Amicus Therapeutics Inc Procédé pour le traitement de la maladie de fabry utilisant des chaperons pharmacologiques
PT3470077T (pt) 2008-02-12 2020-11-30 Amicus Therapeutics Inc Método para previsão da resposta ao tratamento farmacológico de doenças com chaperonas
SG193379A1 (en) * 2011-03-11 2013-10-30 Amicus Therapeutics Inc Dosing regimens for the treatment of fabry disease
WO2014120900A1 (fr) * 2013-01-31 2014-08-07 Icahn School Of Medicine At Mount Sinai Régimes thérapeutiques améliorés pour le traitement de la maladie de fabry
WO2017165164A1 (fr) * 2016-03-22 2017-09-28 Amicus Therapeutics, Inc. Méthodes de traitement de la maladie de fabry chez les patients porteurs de la mutation g9331a au sein du gène gla
TW201806597A (zh) * 2016-07-19 2018-03-01 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
JP2020503900A (ja) * 2017-01-10 2020-02-06 アミカス セラピューティックス インコーポレイテッド ファブリー病の処置のための組換えアルファ−ガラクトシダーゼa
AU2018277756A1 (en) * 2017-05-30 2020-01-23 Amicus Therapeutics, Inc. Methods of treating fabry patients having renal impairment
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
KR20220150902A (ko) * 2020-02-10 2022-11-11 아미쿠스 세라퓨틱스, 인코포레이티드 파브리 질병을 치료하는 방법
AR122390A1 (es) * 2020-03-06 2022-09-07 Amicus Therapeutics Inc Métodos para tratar la enfermedad de fabry en pacientes que tienen una mutación en el gen gla

Also Published As

Publication number Publication date
EP3840753A1 (fr) 2021-06-30
EP4299117A2 (fr) 2024-01-03
EP4285991A3 (fr) 2024-02-21
EP3840753B1 (fr) 2024-07-24
EP4289475A3 (fr) 2024-02-21
EP4285991A2 (fr) 2023-12-06
IL280961A (en) 2021-04-29
MX2021002070A (es) 2021-07-15
EP4289475A2 (fr) 2023-12-13
KR20210046729A (ko) 2021-04-28
JP2023106398A (ja) 2023-08-01
AU2019326252A1 (en) 2021-02-25
KR20240017128A (ko) 2024-02-06
BR112021003137A2 (pt) 2021-05-11
EP4282472A3 (fr) 2024-02-21
US20210315875A1 (en) 2021-10-14
US20240277687A1 (en) 2024-08-22
KR20240017127A (ko) 2024-02-06
EP4299117A3 (fr) 2024-02-21
CL2021000435A1 (es) 2021-10-22
EP4289476A2 (fr) 2023-12-13
CN112888439A (zh) 2021-06-01
JP2021535107A (ja) 2021-12-16
EP4285990A2 (fr) 2023-12-06
CA3110065A1 (fr) 2020-02-27
EP4289476A3 (fr) 2024-02-21
EP4285990A3 (fr) 2024-02-21
EP4282472A2 (fr) 2023-11-29
WO2020040806A1 (fr) 2020-02-27
MX2024008350A (es) 2024-07-19
EA202190569A1 (ru) 2021-06-01

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