Submitted by Anonymous (not verified) on 10 April 2026 - 10:46
Orphan designation: sonpiretigene isteparvovec Treatment of syndromic inherited retinal dystrophies of the rod-dominant phenotype, 22/08/2025 Positive
Source:
Orphan designation: sonpiretigene isteparvovec Treatment of syndromic inherited retinal dystrophies of the rod-dominant phenotype, 22/08/2025 Positive