TW202112372A - 治療具有腎損害患者的Fabry氏病之方法 - Google Patents

治療具有腎損害患者的Fabry氏病之方法 Download PDF

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Publication number
TW202112372A
TW202112372A TW109119640A TW109119640A TW202112372A TW 202112372 A TW202112372 A TW 202112372A TW 109119640 A TW109119640 A TW 109119640A TW 109119640 A TW109119640 A TW 109119640A TW 202112372 A TW202112372 A TW 202112372A
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Taiwan
Prior art keywords
migalastat
patient
frequency
administration
days
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TW109119640A
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English (en)
Chinese (zh)
Inventor
富蘭克林 強森
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美商阿米庫斯醫療股份有限公司
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Publication of TW202112372A publication Critical patent/TW202112372A/zh

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    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01022Alpha-galactosidase (3.2.1.22)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0053Mouth and digestive tract, i.e. intraoral and peroral administration

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Urology & Nephrology (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
TW109119640A 2019-06-11 2020-06-11 治療具有腎損害患者的Fabry氏病之方法 TW202112372A (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962859904P 2019-06-11 2019-06-11
US62/859,904 2019-06-11

Publications (1)

Publication Number Publication Date
TW202112372A true TW202112372A (zh) 2021-04-01

Family

ID=71950725

Family Applications (1)

Application Number Title Priority Date Filing Date
TW109119640A TW202112372A (zh) 2019-06-11 2020-06-11 治療具有腎損害患者的Fabry氏病之方法

Country Status (16)

Country Link
US (1) US20220313670A1 (https=)
EP (1) EP3982962A1 (https=)
JP (2) JP7677910B2 (https=)
KR (1) KR20220019796A (https=)
CN (1) CN114423427A (https=)
AR (1) AR120055A1 (https=)
AU (2) AU2020291002A1 (https=)
BR (1) BR112021024886A2 (https=)
CA (1) CA3141226A1 (https=)
CL (1) CL2021003280A1 (https=)
EA (1) EA202290024A1 (https=)
IL (1) IL288677A (https=)
MX (1) MX2021015352A (https=)
PH (1) PH12021553102A1 (https=)
TW (1) TW202112372A (https=)
WO (1) WO2020252129A1 (https=)

Families Citing this family (7)

* 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
EP2946785B1 (en) 2008-02-12 2018-10-24 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
CA3224546C (en) 2017-05-30 2026-02-24 Amicus Therapeutics, Inc. Use of migalastat in the treatment of fabry patients
KR20200128676A (ko) 2018-02-06 2020-11-16 아미쿠스 세라퓨틱스, 인코포레이티드 임신 환자의 파브리병 치료를 위한 미갈라스타트의 용도
US12594268B2 (en) 2018-08-20 2026-04-07 Amicus Therapeutics, Inc. Methods of treating Fabry disease in patients having a mutation in the GLA gene
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

Family Cites Families (10)

* 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
MX2009010557A (es) 2007-03-30 2009-11-19 Amicus Therapeutics Inc Metodo para el tratamiento de la enfermedad de fabry que utiliza chaperonas farmacologicas.
MX2009011473A (es) * 2007-04-26 2010-01-18 Amicus Therapeutics Inc Regimenes de dosificacion para el tratamiento de enfermedades por almacenamiento lisosomal que utilizan chaperonas farmacologicas.
EP2946785B1 (en) 2008-02-12 2018-10-24 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
SG10201604757RA (en) * 2011-03-11 2016-08-30 Amicus Therapeutics Inc Dosing regimens for the treatment of fabry disease
TW202408513A (zh) * 2016-07-19 2024-03-01 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
WO2018127920A1 (en) * 2017-01-05 2018-07-12 Protalix Ltd. Therapeutic regimen for the treatment of fabry using stabilized alpha-galactosidase
CA3224546C (en) * 2017-05-30 2026-02-24 Amicus Therapeutics, Inc. Use of migalastat in the treatment of fabry patients
AR111971A1 (es) * 2017-05-30 2019-09-04 Amicus Therapeutics Inc Métodos para tratar pacientes con enfermedad de fabry que tienen insuficiencia renal
RS65230B1 (sr) * 2017-05-30 2024-03-29 Amicus Therapeutics Inc Migalastat za lečenje pacijenata koji boluju od fabrijeve bolesti sa oštećenjem bubrega

Also Published As

Publication number Publication date
AU2026200618A1 (en) 2026-04-16
WO2020252129A1 (en) 2020-12-17
IL288677A (en) 2022-02-01
CN114423427A (zh) 2022-04-29
EP3982962A1 (en) 2022-04-20
PH12021553102A1 (en) 2023-10-09
BR112021024886A2 (pt) 2022-01-25
CL2021003280A1 (es) 2022-10-07
KR20220019796A (ko) 2022-02-17
US20220313670A1 (en) 2022-10-06
CA3141226A1 (en) 2020-12-17
JP2025131572A (ja) 2025-09-09
AR120055A1 (es) 2022-02-02
AU2020291002A1 (en) 2022-01-06
MX2021015352A (es) 2022-04-06
JP7677910B2 (ja) 2025-05-15
EA202290024A1 (ru) 2022-03-14
JP2022536687A (ja) 2022-08-18

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