Orphan designation: Adeno-associated viral vector serotype 9 containing the human MECP2 gene, an intron encoding a miRNA generating sequence, and complementary miRNA binding sites Treatment of Rett syndrome, 12/01/2024 Positive

Orphan designation: Adeno-associated viral vector serotype 9 containing the human MECP2 gene, an intron encoding a miRNA generating sequence, and complementary miRNA binding sites Treatment of Rett syndrome, 12/01/2024 Positive