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 of 102 female FD patients from 6 Fabry centers were retrospectively analyzed. According to their FD-specific treatment, patients were separated in 5 groups: Newly agalsidase-beta- [n = 18], agalsidase-alfa- [n = 29] and migalastat-[n = 14] treated patients, and long-term agalsidase-beta- [n = 7] and agalsidase-alfa-[n = 34] treated patients. Clinical presentation and laboratory data, including plasma lyso-Gb₃ levels were assessed.

Results

Treatment with agalsidase-beta,

SUBMITTER: Lenders M 

PROVIDER: S-EPMC11843741 | biostudies-literature | 2025 Feb

REPOSITORIES: biostudies-literature