AR120055A1 - Métodos para tratar la enfermedad de fabry en pacientes que tienen insuficiencia renal - Google Patents

Métodos para tratar la enfermedad de fabry en pacientes que tienen insuficiencia renal

Info

Publication number
AR120055A1
AR120055A1 ARP200101648A ARP200101648A AR120055A1 AR 120055 A1 AR120055 A1 AR 120055A1 AR P200101648 A ARP200101648 A AR P200101648A AR P200101648 A ARP200101648 A AR P200101648A AR 120055 A1 AR120055 A1 AR 120055A1
Authority
AR
Argentina
Prior art keywords
disease
patients
methods
kidney failure
patient
Prior art date
Application number
ARP200101648A
Other languages
English (en)
Inventor
Franklin Johnson
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 AR120055A1 publication Critical patent/AR120055A1/es

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
    • 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

Landscapes

  • 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)

Abstract

Se proporcionan métodos para al tratamiento de la enfermedad de Fabry en un paciente que tiene insuficiencia renal. Determinados métodos comprenden administrar al paciente de aproximadamente 100 mg a aproximadamente 300 mg de equivalente de base libre de migalastat o sal del mismo a una frecuencia mayor de una vez cada dos días, tal como una vez cada cuatro o siete días. Determinados métodos comprenden medir la liso-Gb3 y/o el migalastat en una o más muestras de plasma del paciente.
ARP200101648A 2019-06-11 2020-06-11 Métodos para tratar la enfermedad de fabry en pacientes que tienen insuficiencia renal AR120055A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201962859904P 2019-06-11 2019-06-11

Publications (1)

Publication Number Publication Date
AR120055A1 true AR120055A1 (es) 2022-02-02

Family

ID=71950725

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP200101648A AR120055A1 (es) 2019-06-11 2020-06-11 Métodos para tratar la enfermedad de fabry en pacientes que tienen insuficiencia renal

Country Status (15)

Country Link
US (1) US20220313670A1 (es)
EP (1) EP3982962A1 (es)
JP (1) JP2022536687A (es)
KR (1) KR20220019796A (es)
CN (1) CN114423427A (es)
AR (1) AR120055A1 (es)
AU (1) AU2020291002A1 (es)
BR (1) BR112021024886A2 (es)
CA (1) CA3141226A1 (es)
CL (1) CL2021003280A1 (es)
EA (1) EA202290024A1 (es)
IL (1) IL288677A (es)
MX (1) MX2021015352A (es)
TW (1) TW202112372A (es)
WO (1) WO2020252129A1 (es)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2533050B3 (en) 2006-05-16 2015-06-24 Amicus Therapeutics, Inc. Treatment Options For Fabry Disease
EP2946785B1 (en) 2008-02-12 2018-10-24 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
KR102631805B1 (ko) 2017-05-30 2024-01-31 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

Family Cites Families (9)

* 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
EP2142197A4 (en) 2007-03-30 2010-11-10 Amicus Therapeutics Inc PROCESS FOR THE TREATMENT OF FABRY DISEASE USING PHARMACOLOGICAL CHAPERONS
EP2150254A4 (en) * 2007-04-26 2010-11-10 Amicus Therapeutics Inc DOSAGES FOR THE TREATMENT OF LYSOSOMAL STORAGE DISEASES WITH PHARMACOLOGICAL CHAPTERONES
EP2946785B1 (en) 2008-02-12 2018-10-24 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
CA2829947C (en) * 2011-03-11 2019-01-15 Amicus Therapeutics, Inc. Dosing regimens for the treatment of fabry disease
MX2018007669A (es) * 2016-07-19 2018-09-12 Amicus Therapeutics Inc Tratamiento de la enfermedad de fabry en pacientes no tratados y tratados previamente con ert.
BR112019013920A2 (pt) * 2017-01-05 2020-02-04 Protalix Ltd método para tratar doença de fabry, e, forma unitária de dosagem.
KR102631805B1 (ko) * 2017-05-30 2024-01-31 아미쿠스 세라퓨틱스, 인코포레이티드 신장 손상을 갖는 파브리 환자를 치료하는 방법
AR111971A1 (es) * 2017-05-30 2019-09-04 Amicus Therapeutics Inc Métodos para tratar pacientes con enfermedad de fabry que tienen insuficiencia renal

Also Published As

Publication number Publication date
AU2020291002A1 (en) 2022-01-06
US20220313670A1 (en) 2022-10-06
WO2020252129A1 (en) 2020-12-17
MX2021015352A (es) 2022-04-06
BR112021024886A2 (pt) 2022-01-25
IL288677A (en) 2022-02-01
CA3141226A1 (en) 2020-12-17
CN114423427A (zh) 2022-04-29
CL2021003280A1 (es) 2022-10-07
KR20220019796A (ko) 2022-02-17
EA202290024A1 (ru) 2022-03-14
TW202112372A (zh) 2021-04-01
JP2022536687A (ja) 2022-08-18
EP3982962A1 (en) 2022-04-20

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