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)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Urology & Nephrology (AREA)
  • Cardiology (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)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Analytical Chemistry (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Biophysics (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Pathology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (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 (en) 2018-08-20 2019-01-16 Methods of treating fabry disease in patients having a mutation in the gla gene

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 (1) US20210315875A1 (ko)
EP (7) EP4285990A3 (ko)
JP (2) JP2021535107A (ko)
KR (3) KR20210046729A (ko)
CN (1) CN112888439A (ko)
AU (1) AU2019326252A1 (ko)
BR (1) BR112021003137A2 (ko)
CA (1) CA3110065A1 (ko)
CL (1) CL2021000435A1 (ko)
EA (1) EA202190569A1 (ko)
IL (1) IL280961A (ko)
MX (1) MX2021002070A (ko)
SG (1) SG11202101117QA (ko)
WO (1) WO2020040806A1 (ko)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL2533050T6 (pl) 2006-05-16 2016-05-31 Amicus Therapeutics Inc Możliwości leczenia choroby Fabry'ego
EP2252313B1 (en) 2008-02-12 2015-04-08 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
KR20240017111A (ko) 2017-05-30 2024-02-06 아미쿠스 세라퓨틱스, 인코포레이티드 신장 손상을 갖는 파브리 환자를 치료하는 방법
WO2019157047A1 (en) 2018-02-06 2019-08-15 Amicus Therapeutics, Inc. Use of migalastat for treating fabry disease in pregnant patients
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
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
AU2008232614A1 (en) 2007-03-30 2008-10-09 Amicus Therapeutics, Inc. Method for the treatment of Fabry disease using pharmacological chaperones
EP2252313B1 (en) 2008-02-12 2015-04-08 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
EP2683382B1 (en) * 2011-03-11 2020-04-22 Amicus Therapeutics, Inc. Dosing regimens for the treatment of fabry disease
WO2014120900A1 (en) * 2013-01-31 2014-08-07 Icahn School Of Medicine At Mount Sinai Enhanced therapeutic regimens for treating fabry disease
CN114159436A (zh) * 2016-03-22 2022-03-11 阿米库斯治疗学公司 治疗在gla基因中具有g9331a突变的患者的法布里病的方法
US20190183869A1 (en) * 2016-07-19 2019-06-20 Amicus Therapeutics, Inc. Treatment of fabry disease in ert-naïve and ert-experienced patients
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
KR20240017111A (ko) * 2017-05-30 2024-02-06 아미쿠스 세라퓨틱스, 인코포레이티드 신장 손상을 갖는 파브리 환자를 치료하는 방법
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
EP4103178A4 (en) * 2020-02-10 2024-03-20 Amicus Therapeutics Inc METHODS OF TREATMENT OF FABRY’S DISEASE
TW202146020A (zh) * 2020-03-06 2021-12-16 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

Also Published As

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

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