Posted inHematology-Oncology news Pediatrics
Beyond the CNS and Skeleton: HSPC-Gene Therapy (OTL-203) Demonstrates Superior Multi-System Efficacy in Hurler Syndrome
A clinical study comparing OTL-203 gene therapy to allogeneic HSCT for Hurler syndrome reveals significant improvements in corneal clarity, hearing preservation, and cardiac stability, marking a potential shift in the standard of care for multisystemic lysosomal storage disorders.