Browsing Tag
pediatric neurology
2 posts
Neurogene (NASDAQ: NGNE) advances Rett syndrome gene therapy with Embolden trial launch and positive pediatric data
Neurogene reports progress on NGN-401 Rett syndrome gene therapy with pivotal trial launch and strong pediatric data. See what’s ahead in 2026.
JR-446 developed by JCR Pharmaceuticals secures orphan drug designation from the European Commission for MPS IIIB
The European Commission has granted orphan drug status to JR-446 for MPS IIIB, supporting JCR Pharmaceuticals’ global rare disease expansion.