Impact of enzyme replacement therapy and migalastat on disease progression in females with fabry disease

Impact of enzyme replacement therapy and migalastat on disease progression in females with fabry disease

Abstract Aim The aim of our multicenter study was to investigate the safety and efficacy of enzyme replacement therapy (ERT) and chaperone therapy on the disease progression in female Fabry disease (FD) patients and to compare the individual treatment regimens. Methods Data from 3 consecutive visits...

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Bibliographic Details
Main Authors: Malte Lenders, Albina Nowak, Markus Cybulla, Jessica Kaufeld, Anja Friederike Köhn, Nicole Maria Muschol, Christine Kurschat, Eva Brand
Format: Article
Language:English
Published: BMC 2025-02-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Females
Fabry disease
Enzyme replacement therapy
Migalastat
Disease progression
Online Access:https://doi.org/10.1186/s13023-025-03600-y
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https://doi.org/10.1186/s13023-025-03600-y

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