KR20240034203A - 소아청소년 환자에서 파브리병을 치료하는 방법 - Google Patents

소아청소년 환자에서 파브리병을 치료하는 방법 Download PDF

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Publication number
KR20240034203A
KR20240034203A KR1020247003776A KR20247003776A KR20240034203A KR 20240034203 A KR20240034203 A KR 20240034203A KR 1020247003776 A KR1020247003776 A KR 1020247003776A KR 20247003776 A KR20247003776 A KR 20247003776A KR 20240034203 A KR20240034203 A KR 20240034203A
Authority
KR
South Korea
Prior art keywords
migalastat
patient
years
therapeutically effective
effective dose
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
KR1020247003776A
Other languages
English (en)
Korean (ko)
Inventor
프랑클린 존슨
Original Assignee
아미쿠스 세라퓨틱스, 인코포레이티드
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 아미쿠스 세라퓨틱스, 인코포레이티드 filed Critical 아미쿠스 세라퓨틱스, 인코포레이티드
Publication of KR20240034203A publication Critical patent/KR20240034203A/ko
Pending legal-status Critical Current

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic 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
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7008—Compounds having an amino group directly attached to a carbon atom of the saccharide radical, e.g. D-galactosamine, ranimustine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • Molecular Biology (AREA)
  • Diabetes (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
KR1020247003776A 2021-07-12 2022-07-12 소아청소년 환자에서 파브리병을 치료하는 방법 Pending KR20240034203A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163220816P 2021-07-12 2021-07-12
US63/220,816 2021-07-12
PCT/US2022/073626 WO2023288210A1 (en) 2021-07-12 2022-07-12 Methods of treating fabry disease in pediatric patients

Publications (1)

Publication Number Publication Date
KR20240034203A true KR20240034203A (ko) 2024-03-13

Family

ID=83149437

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020247003776A Pending KR20240034203A (ko) 2021-07-12 2022-07-12 소아청소년 환자에서 파브리병을 치료하는 방법

Country Status (7)

Country Link
US (1) US20240293387A1 (https=)
EP (1) EP4370120A1 (https=)
JP (1) JP2024525760A (https=)
KR (1) KR20240034203A (https=)
AU (1) AU2022310691A1 (https=)
CA (1) CA3225511A1 (https=)
WO (1) WO2023288210A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3851105B1 (en) 2019-08-07 2026-03-04 Amicus Therapeutics, Inc. Migalastat for use in treating fabry disease in patients having a mutation in the gla gene

Family Cites Families (3)

* 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
SI3470077T1 (sl) 2008-02-12 2020-12-31 Amicus Therapeutics, Inc. Postopek za napovedovanje odgovora na zdravljenje bolezni s farmakološkim spremljevalcem

Also Published As

Publication number Publication date
AU2022310691A1 (en) 2024-01-25
EP4370120A1 (en) 2024-05-22
JP2024525760A (ja) 2024-07-12
WO2023288210A1 (en) 2023-01-19
CA3225511A1 (en) 2023-01-19
US20240293387A1 (en) 2024-09-05

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PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

R18-X000 Changes to party contact information recorded

St.27 status event code: A-3-3-R10-R18-oth-X000

D11 Substantive examination requested

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D11-EXM-PA0201 (AS PROVIDED BY THE NATIONAL OFFICE)

PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201