Long-term efficacy of migalastat in females with Fabry disease.

ABSTRACT:

Background

Fabry disease is a progressive, X-linked lysosomal disorder caused by reduced or absent α-galactosidase A activity due to GLA variants. Females with Fabry disease often experience diagnostic delays and an underappreciated disease burden owing to their variable disease presentation and progression.

Methods

We conducted a post hoc analysis of all females from the clinical studies FACETS (NCT00925301) and ATTRACT (NCT01218659) and their open-label extensions, assessing baseline characteristics and long-term efficacy of migalastat regarding cardiac and renal function and Fabry-associated clinical events (FACEs).

Results

Overall, 60 females had a median migalastat exposure of 5.1 years. At baseline, the median age was 47 years with multiorgan invol

SUBMITTER: Kallish S 

PROVIDER: S-EPMC12703307 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature