Enzyme replacement therapy in infants and very young children with Gaucher disease using velaglucerase alfa: a single-center experience
These preliminary data suggest that velaglucerase alfa is well-tolerated and associated with improvements in clinical parameters in very young children with GD types 1 and 3, offering insights into the early presentation and course of GD in infancy and early childhood.
Authors
- Ravi Ramesh Pathak (ORCID: https://orcid.org/0000-0002-9689-6475)
- Ekaterina Wright
- Özlem Göker-Alpan (ORCID: https://orcid.org/0000-0003-4758-6793)
- Margarita M. Ivanova (ORCID: https://orcid.org/0000-0002-6197-8606)
Institutions
- Lysosomal and Rare Disorders Research and Treatment Center (US)
- Takeda (United States) (US)
Publication Details
- Journal
- Frontiers in Pediatrics
- Published
- 2025-10-17
- DOI
- https://doi.org/10.3389/fped.2025.1613599
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00