Orphan designation: Adeno-associated viral vector serotype 2i8 containing the human LAMP2 isoform B transgene Treatment of Danon disease, 22/05/2025 Positive
Orphan designation: Adeno-associated viral vector serotype 2i8 containing the human LAMP2 isoform B transgene Treatment of Danon disease, 22/05/2025 Positive
