Articles from December 2024

Follow-Up Results of First Approved Drug Therapy for Inherited Diseases
Monday, December 9, 2024
Dr. Stephen Russell recently shared the 8 and 9-year follow-up results of the phase 3 trial of voretigene neparvovec-rzyl (Luxturna) at the American Society of Retina Specialists (ASRS) meeting. This gene therapy, the first approved for any inherited disease, targets a specific type of retinal dystrophy caused by RPE65 mutations.