Real-world outcomes of voretigene neparvovec treatment in pediatric patients with RPE65-associated Leber congenital amaurosis
Callie Deng, Peter Y. Zhao, Kari Branham, Dana Schlegel, Abigail T. Fahim, Thiran K. Jayasundera, Naheed Khan, Cagri G. Besirli
Topics & Concepts
MedicinePediatricsClinical trialVisual acuityMEDLINECongenital diseaseEye diseaseRandomized controlled trialLongitudinal studyDiseaseRetinal Development and DisordersRetinal Diseases and TreatmentsRetinopathy of Prematurity Studies