Outcome of enzyme replacement therapy for hematological and visceral manifestations in children with acid sphingomyelinase deficiency: a single center experience in upper Egypt
Our findings indicate that olipudase alfa provides significant benefits in key hematological and visceral clinical outcomes in pediatric patients with ASMD.
Authors
- Esraa Hefzy Shaker
- Mervat A. M. Youssef (ORCID: https://orcid.org/0000-0002-9054-0662)
- N. Saleh
Institutions
- Mediclinic City Hospital (AE)
- Assiut University (EG)
Publication Details
- Journal
- Molecular and Cellular Pediatrics
- Published
- 2025-08-14
- DOI
- https://doi.org/10.1186/s40348-025-00199-9
- Citations
- 1
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 1.16
Funders
- Long Island University
- Science and Technology Development Fund