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.

Conditions

• Leber Congenital Amaurosis 10
• Blindness
• Leber Congenital Amaurosis
• Vision Disorders
• Sensation Disorders
• Neurologic Manifestations
• Eye Diseases
• Eye Disorders Congenital
• Retinal Disease
• Retinal Degeneration
• Retinal Dystrophies

Interventions

• DRUG: sepofarsen

Sponsor

ProQR Therapeutics