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

Orphanet J Rare Dis · 2025 · PMC11843741 · PMID 39980015

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showed a trendno p-value reported

Females newly treated with agalsidase-beta showed a trend for a reduction of GI symptoms, whereas those newly treated with agalsidase-alfa showed a trend for a reduction of FD-related pain.

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