AR122298A1 - METHODS TO TREAT FABRY DISEASE IN PATIENTS WHO HAVE A MUTATION IN THE GLA GENE - Google Patents

METHODS TO TREAT FABRY DISEASE IN PATIENTS WHO HAVE A MUTATION IN THE GLA GENE

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Publication number
AR122298A1
AR122298A1 ARP200102261A ARP200102261A AR122298A1 AR 122298 A1 AR122298 A1 AR 122298A1 AR P200102261 A ARP200102261 A AR P200102261A AR P200102261 A ARP200102261 A AR P200102261A AR 122298 A1 AR122298 A1 AR 122298A1
Authority
AR
Argentina
Prior art keywords
fabry disease
methods
mutation
patients
galactosidase
Prior art date
Application number
ARP200102261A
Other languages
Spanish (es)
Inventor
Elfrida Benjamin
Xiaoyang Wu
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 AR122298A1 publication Critical patent/AR122298A1/en

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Se proporcionan métodos para tratar un paciente diagnosticado con la enfermedad de Fabry y métodos para mejorar la a-galactosidasa A en un paciente diagnosticado o con sospecha de padecer la enfermedad de Fabry. Algunos métodos comprenden administrar a un paciente una dosis terapéuticamente eficaz de una chaperona farmacológica para a-galactosidasa A, donde el paciente tiene una mutación en la secuencia de ácido nucleico que codifica la a-galactosidasa A. También se describen usos de chaperonas farmacológicas para el tratamiento de la enfermedad de Fabry y composiciones para su uso en el tratamiento de la enfermedad de Fabry.Methods of treating a patient diagnosed with Fabry disease and methods of improving α-galactosidase A in a patient diagnosed with or suspected of having Fabry disease are provided. Some methods comprise administering to a patient a therapeutically effective dose of a pharmacological chaperone for α-galactosidase A, where the patient has a mutation in the nucleic acid sequence encoding α-galactosidase A. Uses of pharmacological chaperones for the treatment of Fabry disease and compositions for use in the treatment of Fabry disease.

ARP200102261A 2019-08-07 2020-08-07 METHODS TO TREAT FABRY DISEASE IN PATIENTS WHO HAVE A MUTATION IN THE GLA GENE AR122298A1 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201962883756P 2019-08-07 2019-08-07

Publications (1)

Publication Number Publication Date
AR122298A1 true AR122298A1 (en) 2022-08-31

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
ARP200102261A AR122298A1 (en) 2019-08-07 2020-08-07 METHODS TO TREAT FABRY DISEASE IN PATIENTS WHO HAVE A MUTATION IN THE GLA GENE

Country Status (1)

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AR (1) AR122298A1 (en)

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