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HUE068783T2 - Migalasztát a Fabry-kór kezelésére szolgáló módszerekben való felhasználására olyan betegeknél, akiknél a GLA génben mutáció található - Google Patents

Migalasztát a Fabry-kór kezelésére szolgáló módszerekben való felhasználására olyan betegeknél, akiknél a GLA génben mutáció található

Info

Publication number
HUE068783T2
HUE068783T2 HUE19703514A HUE19703514A HUE068783T2 HU E068783 T2 HUE068783 T2 HU E068783T2 HU E19703514 A HUE19703514 A HU E19703514A HU E19703514 A HUE19703514 A HU E19703514A HU E068783 T2 HUE068783 T2 HU E068783T2
Authority
HU
Hungary
Prior art keywords
migalastat
mutation
patients
methods
fabry disease
Prior art date
Application number
HUE19703514A
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=65279726&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=HUE068783(T2) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Amicus Therapeutics Inc filed Critical Amicus Therapeutics Inc
Publication of HUE068783T2 publication Critical patent/HUE068783T2/hu

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)
  • 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)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Pathology (AREA)
  • Biochemistry (AREA)
HUE19703514A 2018-08-20 2019-01-16 Migalasztát a Fabry-kór kezelésére szolgáló módszerekben való felhasználására olyan betegeknél, akiknél a GLA génben mutáció található HUE068783T2 (hu)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201862719962P 2018-08-20 2018-08-20

Publications (1)

Publication Number Publication Date
HUE068783T2 true HUE068783T2 (hu) 2025-01-28

Family

ID=65279726

Family Applications (1)

Application Number Title Priority Date Filing Date
HUE19703514A HUE068783T2 (hu) 2018-08-20 2019-01-16 Migalasztát a Fabry-kór kezelésére szolgáló módszerekben való felhasználására olyan betegeknél, akiknél a GLA génben mutáció található

Country Status (25)

Country Link
US (2) US20210315875A1 (hu)
EP (7) EP4285991A3 (hu)
JP (3) JP2021535107A (hu)
KR (3) KR20210046729A (hu)
CN (1) CN112888439A (hu)
AU (2) AU2019326252A1 (hu)
BR (1) BR112021003137A2 (hu)
CA (1) CA3110065A1 (hu)
CL (3) CL2021000435A1 (hu)
DK (1) DK3840753T3 (hu)
EA (1) EA202190569A1 (hu)
ES (1) ES2993833T3 (hu)
FI (1) FI3840753T3 (hu)
HR (1) HRP20241454T1 (hu)
HU (1) HUE068783T2 (hu)
IL (1) IL280961A (hu)
LT (1) LT3840753T (hu)
MX (2) MX2021002070A (hu)
PL (1) PL3840753T3 (hu)
PT (1) PT3840753T (hu)
RS (1) RS66094B1 (hu)
SG (1) SG11202101117QA (hu)
SI (1) SI3840753T1 (hu)
SM (1) SMT202400436T1 (hu)
WO (1) WO2020040806A1 (hu)

Families Citing this family (9)

* 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
DK3470077T3 (da) 2008-02-12 2020-11-30 Amicus Therapeutics Inc Fremgangsmåde til forudsigelse af respons på behandling af sygdomme med farmakologiske chaperoner
CA3224546A1 (en) 2017-05-30 2018-12-06 Jeff Castelli Use of migalastat in the treatment of fabry patients having renal impairment
JP7555818B2 (ja) 2018-02-06 2024-09-25 アミカス セラピューティックス インコーポレイテッド 妊娠患者におけるファブリー病の治療のためのミガラスタットの使用
KR20220044560A (ko) 2019-08-07 2022-04-08 아미쿠스 세라퓨틱스, 인코포레이티드 Gla 유전자에 돌연변이를 갖는 환자에서 파브리 질병을 치료하는 방법
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
CN116656704B (zh) * 2023-06-01 2024-11-05 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 (14)

* 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
DK3470077T3 (da) * 2008-02-12 2020-11-30 Amicus Therapeutics Inc Fremgangsmåde til forudsigelse af respons på behandling af sygdomme med farmakologiske chaperoner
SG193379A1 (en) * 2011-03-11 2013-10-30 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
IL313907A (en) * 2016-07-19 2024-08-01 Amicus Therapeutics Inc Treatment of Fabry disease in patients experienced with ERT-NAIVE and ERT
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
CA3224546A1 (en) * 2017-05-30 2018-12-06 Jeff Castelli Use of migalastat in the treatment of fabry patients having renal impairment
JP7555818B2 (ja) * 2018-02-06 2024-09-25 アミカス セラピューティックス インコーポレイテッド 妊娠患者におけるファブリー病の治療のためのミガラスタットの使用
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
KR20220044560A (ko) * 2019-08-07 2022-04-08 아미쿠스 세라퓨틱스, 인코포레이티드 Gla 유전자에 돌연변이를 갖는 환자에서 파브리 질병을 치료하는 방법
AU2021218578A1 (en) * 2020-02-10 2022-08-25 Amicus Therapeutics, Inc. Methods of treating Fabry disease
PL4114390T3 (pl) * 2020-03-06 2025-06-23 Amicus Therapeutics, Inc. Sposoby leczenia choroby fabry’ego u pacjentów z mutacją w genie gla

Also Published As

Publication number Publication date
FI3840753T3 (fi) 2024-10-30
AU2019326252A1 (en) 2021-02-25
US20210315875A1 (en) 2021-10-14
CL2024000739A1 (es) 2024-10-04
HRP20241454T1 (hr) 2024-12-20
CA3110065A1 (en) 2020-02-27
EP4282472A2 (en) 2023-11-29
JP7784398B2 (ja) 2025-12-11
PL3840753T3 (pl) 2025-01-20
DK3840753T3 (da) 2024-10-28
SG11202101117QA (en) 2021-03-30
EP4289476A3 (en) 2024-02-21
EP4289476A2 (en) 2023-12-13
SMT202400436T1 (it) 2024-11-15
EP4289475A3 (en) 2024-02-21
JP2023106398A (ja) 2023-08-01
LT3840753T (lt) 2024-11-11
SI3840753T1 (sl) 2025-01-31
MX2021002070A (es) 2021-07-15
CL2024000741A1 (es) 2024-10-04
KR20240017128A (ko) 2024-02-06
EP4285991A3 (en) 2024-02-21
MX2024008350A (es) 2024-07-19
EA202190569A1 (ru) 2021-06-01
WO2020040806A1 (en) 2020-02-27
EP3840753B1 (en) 2024-07-24
EP4285990A3 (en) 2024-02-21
CL2021000435A1 (es) 2021-10-22
EP4285991A2 (en) 2023-12-06
AU2025205141A1 (en) 2025-07-31
US20240277687A1 (en) 2024-08-22
CN112888439A (zh) 2021-06-01
EP4289475A2 (en) 2023-12-13
KR20240017127A (ko) 2024-02-06
EP4282472A3 (en) 2024-02-21
ES2993833T3 (en) 2025-01-10
EP4299117A3 (en) 2024-02-21
JP2021535107A (ja) 2021-12-16
RS66094B1 (sr) 2024-11-29
PT3840753T (pt) 2024-10-30
JP2025165955A (ja) 2025-11-05
KR20210046729A (ko) 2021-04-28
EP3840753A1 (en) 2021-06-30
EP4299117A2 (en) 2024-01-03
IL280961A (en) 2021-04-29
EP4285990A2 (en) 2023-12-06
BR112021003137A2 (pt) 2021-05-11

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