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February 22, 2026Open Access

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

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Authors

MLMalte LendersANAlbina NowakMCMarkus Cybulla

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Overview

Multicenter study investigates treatment effects on disease progression in females with Fabry disease, indicating overall safety and efficacy.

Key Points

  • To assess the safety and efficacy of enzyme replacement therapy and migalastat in female patients with Fabry disease.
  • Retrospective analysis of data from 102 female Fabry disease patients across 6 centers.
  • Patients grouped by treatment: agalsidase-beta, agalsidase-alfa, and migalastat.
  • Clinical presentations and laboratory data, including plasma lyso-Gb levels, were assessed during three visits.
  • Treatment with all therapies was safe, with rare severe adverse events.
  • Patients exhibited a stable disease course, with no hospitalizations due to cardiac issues.
  • eGFR slightly decreased in agalsidase-alfa patients but remained within normal decline range; no significant changes in agalsidase-beta or migalastat groups.

Cite This Study

Lenders et al. (2025) studied this question.

synapsesocial.com/papers/699a9dae482488d673cd3b60https://doi.org/10.5167/uzh-292071
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Clinical outcomes in Fabry Patients switching to agalsidase beta for renal ineffectiveness of the primary Fabry therapy: a single-center analysis2025
  2. 2Long-term efficacy of migalastat in females with Fabry disease2025 · 2 citations
  3. 3Safety and efficacy of migalastat in Fabry's disease treatment with cardiac impact : a systematic review and meta-analysis2025
  4. 4Impact of enzyme replacement therapy and migalastat on left atrial strain and cardiomyopathy in patients with Fabry disease2023 · 9 citations
  5. 5FollowME Fabry Pathfinders real-world registry in Spain and Portugal: cardiac and renal outcomes with migalastat in patients with Fabry disease2025