Yes, indeed, the FDA Advisory Committee Unanimously Recommends Approval of Investigational LUXTURNA™ (voretigene neparvovec) for Patients with Biallelic RPE65-mediated Inherited Retinal Disease
LUXTURNA has the potential to be the first pharmacologic treatment for an inherited retinal disease (IRD) and the first gene therapy for a genetic disease in the United State
First adeno-associated viral (AAV . . .