Clinical outcomes of exclusive enzyme therapy (laronidase) in a cohort of patients with mucopolysaccharidosis type I
Early ERT and regular multidisciplinary management are effective in slowing disease progression in severe and attenuated patients with MPS I and helping to maintain autonomy in patients with attenuated MPS I, ensuring a better quality of life.
Authors
- Eliane Le Peillet Feuillet
- Magali Pettazzoni (ORCID: https://orcid.org/0000-0002-9569-912X)
- Nathalie Reynes
- P. Journeau (ORCID: https://orcid.org/0000-0001-7709-4429)
- Nathalie Guffon (ORCID: https://orcid.org/0000-0002-3052-2366)
- Nicolas Pangaud
- Alain Fouilhoux
Institutions
- Sanofi (France) (FR)
- Hôpital Femme Mère Enfant (FR)
- Hospices Civils de Lyon (FR)
- Hôpital Louis Pradel (FR)
Publication Details
- Journal
- Orphanet Journal of Rare Diseases
- Published
- 2025-12-06
- DOI
- https://doi.org/10.1186/s13023-025-04157-6
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00