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July 19, 2024Journal of Inherited Metabolic DiseaseOpen Access

Renal and multisystem effectiveness of 3.9 years of migalastat in a global real‐world cohort: Results from the followME Fabry Pathfinders registry

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Authors

DHDerralynn HughesRoyal Free London NHS Foundation TrustGSGere Sunder–PlassmannMedical University of ViennaAJAna JovanovićManchester Academic Health Science Centre

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Cite This Study

Hughes et al. (2024) studied this question.

synapsesocial.com/papers/68e5fb6fb6db64358758efa2https://doi.org/10.1002/jimd.12771
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Also Consider

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

  1. 1FollowME Fabry Pathfinders real-world registry in Spain and Portugal: cardiac and renal outcomes with migalastat in patients with Fabry disease2025
  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. 4Fabry disease Enzyme Enhancement on migalastat Study: FEES2024 · 2 citations
  5. 5Impact of enzyme replacement therapy and migalastat on disease progression in females with fabry disease2025