12-month outcomes after voretigene neparvovec gene therapy in paediatric patients with RPE65-mediated inherited retinal dystrophy.

چکیده

To report main outcomes and complications following voretigene neparvovec (Luxturna) treatment in paediatric patients. Records of patients under the age of 17 treated by subretinal administration of voretigene neparvovec for confirmed biallelic RPE65-mediated inherited retinal dystrophy were…

متن کامل چکیده در سایت ناشر در دسترس است.

موضوعات مرتبط

• تصویربرداری و تشخیص