Real-world treatment with elosulfase alfa in patients with MPS IVA is associated with improved endurance over time
This analysis assessed associations between ERT exposure and endurance to confirm the real-world, long-term effectiveness of elosulfase alfa in participants with MPS IVA.
Authors
- Karolina M. Stępień (ORCID: https://orcid.org/0000-0003-0148-6332)
- Pascal Reisewitz
- Abigail Hunt
- David A. Hinds (ORCID: https://orcid.org/0000-0002-4911-803X)
- Philippe M. Campeau (ORCID: https://orcid.org/0000-0001-9713-7107)
- Barbara K. Burton
- Jaim Sutton
Institutions
- Lurie Children's Hospital (US)
- Centre Hospitalier Universitaire Sainte-Justine (CA)
- Northern Health and Social Care Trust (GB)
- BioMarin (United Kingdom) (GB)
- BioMarin (United States) (US)
Publication Details
- Journal
- Genetics in Medicine Open
- Published
- 2025-01-01
- DOI
- https://doi.org/10.1016/j.gimo.2025.103428
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00