Submitted by Anonymous (not verified) on 14 November 2024 - 11:03
Orphan designation: adeno-associated virus vector serotype SAN011 encoding a microRNA against DMPK mRNA Treatment of dystrophic myotonia type 1, 11/10/2024 Positive
Source:
Orphan designation: adeno-associated virus vector serotype SAN011 encoding a microRNA against DMPK mRNA Treatment of dystrophic myotonia type 1, 11/10/2024 Positive