Long-term enzyme replacement therapy: Findings from the mucopolysaccharidosis VI clinical surveillance program after 15 years follow-up
Data collected in the CSP over 15 years provide real-world evidence for sustained improvements in endurance and pulmonary function among patients with MPS VI treated with ERT, with no new safety concerns identified. These results further support and confirm observations from the clinical trials and previous findings from the CSP.
Authors
- Paul Harmatz (ORCID: https://orcid.org/0000-0001-5603-2085)
- Christina Lampe (ORCID: https://orcid.org/0000-0003-4953-7119)
- Veronika Horváthová
- Elisa Leão Teles (ORCID: https://orcid.org/0000-0002-3011-0249)
- Rossella Parini (ORCID: https://orcid.org/0000-0003-4505-1306)
- Reena Sharma (ORCID: https://orcid.org/0000-0002-5417-2815)
- Barbara K. Burton
- Alice Lail
Institutions
- Universitätsklinikum Gießen und Marburg (DE)
- Salford Royal NHS Foundation Trust (GB)
- Hospital de São João (PT)
- BioMarin (United Kingdom) (GB)
- Salford Royal Hospital (GB)
- UCSF Benioff Children's Hospital (US)
- The San Raffaele Telethon Institute for Gene Therapy (IT)
- BioMarin (United States) (US)
Publication Details
- Journal
- Molecular Genetics and Metabolism
- Published
- 2025-05-08
- DOI
- https://doi.org/10.1016/j.ymgme.2025.109135
- Citations
- 1
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 1.16
Funders
- BioMarin Pharmaceutical