Long-term efficacy of migalastat in females with Fabry disease
These data show that females with Fabry disease experience considerable disease severity and burden and support the long-term efficacy of migalastat for the treatment of females.
Authors
- Robert J. Hopkin (ORCID: https://orcid.org/0000-0003-0770-5516)
- Roser Torrá (ORCID: https://orcid.org/0000-0001-8714-2332)
- Peter Nordbeck (ORCID: https://orcid.org/0000-0002-2560-4068)
- Antonia Camporeale (ORCID: https://orcid.org/0000-0001-7604-0496)
- Ana Jovanović (ORCID: https://orcid.org/0000-0002-1523-6580)
- Staci Kallish (ORCID: https://orcid.org/0000-0003-2515-2097)
- Eva Krusinska
- Biliana O. Veleva-Rotse (ORCID: https://orcid.org/0000-0002-7783-0112)
Institutions
- Cincinnati Children's Hospital Medical Center (US)
- Manchester University NHS Foundation Trust (GB)
- IRCCS Policlinico San Donato (IT)
- Translational Therapeutics (United States) (US)
- Universitätsklinikum Würzburg (DE)
- Amicus Therapeutics (United States) (US)
- Puigvert Foundation (ES)
- University of Pennsylvania (US)
- University of Cincinnati Medical Center (US)
Publication Details
- Journal
- Journal of Medical Genetics
- Published
- 2025-09-02
- DOI
- https://doi.org/10.1136/jmg-2024-110596
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00