CN115427040A - 治疗在gla基因中具有突变的患者的法布里病的方法 - Google Patents

治疗在gla基因中具有突变的患者的法布里病的方法 Download PDF

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Publication number
CN115427040A
CN115427040A CN202180028403.5A CN202180028403A CN115427040A CN 115427040 A CN115427040 A CN 115427040A CN 202180028403 A CN202180028403 A CN 202180028403A CN 115427040 A CN115427040 A CN 115427040A
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mutation
patient
migalastat
seq
gal
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Pending
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CN202180028403.5A
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Chinese (zh)
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埃尔弗里达·本杰明
吴晓阳
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Amicus Therapeutics Inc
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Amicus Therapeutics Inc
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Publication of CN115427040A publication Critical patent/CN115427040A/zh
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    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/106—Pharmacogenomics, i.e. genetic variability in individual responses to drugs and drug metabolism
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/156—Polymorphic or mutational markers

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  • 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)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • Analytical Chemistry (AREA)
  • Zoology (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Pathology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
CN202180028403.5A 2020-03-06 2021-03-05 治疗在gla基因中具有突变的患者的法布里病的方法 Pending CN115427040A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062986297P 2020-03-06 2020-03-06
US62/986,297 2020-03-06
PCT/US2021/020984 WO2021178737A1 (en) 2020-03-06 2021-03-05 Methods of treating fabry disease in patients having a mutation in the gla gene

Publications (1)

Publication Number Publication Date
CN115427040A true CN115427040A (zh) 2022-12-02

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CN202180028403.5A Pending CN115427040A (zh) 2020-03-06 2021-03-05 治疗在gla基因中具有突变的患者的法布里病的方法

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Country Link
US (1) US20230136297A1 (https=)
EP (2) EP4114390B1 (https=)
JP (2) JP7749572B2 (https=)
KR (1) KR20220152547A (https=)
CN (1) CN115427040A (https=)
AR (1) AR122390A1 (https=)
AU (1) AU2021230371A1 (https=)
BR (1) BR112022017881A2 (https=)
CA (1) CA3170718A1 (https=)
CL (1) CL2022002423A1 (https=)
DK (1) DK4114390T3 (https=)
ES (1) ES3013813T3 (https=)
FI (1) FI4114390T3 (https=)
HR (1) HRP20250179T1 (https=)
HU (1) HUE070116T2 (https=)
IL (1) IL296221A (https=)
LT (1) LT4114390T (https=)
MX (1) MX2022011030A (https=)
PL (1) PL4114390T3 (https=)
PT (1) PT4114390T (https=)
RS (1) RS66530B1 (https=)
SI (1) SI4114390T1 (https=)
SM (1) SMT202500072T1 (https=)
TW (1) TW202146020A (https=)
WO (1) WO2021178737A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4282472A3 (en) * 2018-08-20 2024-02-21 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having a mutation in the gla gene
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

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009102895A2 (en) * 2008-02-12 2009-08-20 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases

Family Cites Families (2)

* 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

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009102895A2 (en) * 2008-02-12 2009-08-20 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
D.P. GERMAIN等: "Treatment of Fabry\'s Disease with the Pharmacologic Chaperone Migalastat", THE NEW ENGLAND JOURNAL OF MEDICINE, vol. 375, no. 6, 11 August 2016 (2016-08-11), pages 545 - 555 *
ELFRIDA R. BENJAMIN等: "The validation of pharmacogenetics for the identification of Fabry patients to be treated with migalastat", GENETICS IN MEDICINE, vol. 19, no. 4, 22 September 2016 (2016-09-22), pages 430 - 438, XP055582063, DOI: 10.1038/gim.2016.122 *

Also Published As

Publication number Publication date
TW202146020A (zh) 2021-12-16
RS66530B1 (sr) 2025-03-31
AR122390A1 (es) 2022-09-07
EP4114390A1 (en) 2023-01-11
EP4114390B1 (en) 2024-12-04
JP2023516753A (ja) 2023-04-20
LT4114390T (lt) 2025-02-10
US20230136297A1 (en) 2023-05-04
WO2021178737A1 (en) 2021-09-10
SMT202500072T1 (it) 2025-03-12
BR112022017881A2 (pt) 2022-12-06
MX2022011030A (es) 2022-12-13
JP7749572B2 (ja) 2025-10-06
AU2021230371A1 (en) 2022-09-29
KR20220152547A (ko) 2022-11-16
CA3170718A1 (en) 2021-09-10
FI4114390T3 (fi) 2025-03-07
IL296221A (en) 2022-11-01
CL2022002423A1 (es) 2023-05-05
PL4114390T3 (pl) 2025-06-23
PT4114390T (pt) 2025-02-24
EP4527454A2 (en) 2025-03-26
HRP20250179T1 (hr) 2025-04-11
ES3013813T3 (en) 2025-04-15
JP2026016375A (ja) 2026-02-03
EP4527454A3 (en) 2025-09-24
SI4114390T1 (sl) 2025-03-31
HUE070116T2 (hu) 2025-05-28
DK4114390T3 (da) 2025-02-24

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