Leber Congenital Amaurosis

Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)

Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2

Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

Inherited Retinal Degenerative Disease Registry

Extension Study to Study PQ-110-001 (NCT03140969)

Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT)

Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)

Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D

Natural History Study of Patients With Leber Congenital Amaurosis Associated With Mutations in RPE65

An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.

A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)

Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)

Safety Study in Subjects With Leber Congenital Amaurosis

Clinical Trial of Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations

Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis

Genetic Decryption of Leber Congenital Amaurosis (LCA) in a Large Cohort of Independent Families.

Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65