Clinical Trial Update: 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. [Phase 2, Phase 3]

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Summary

An open-label dose-escalation study with a double-masked, randomized design is evaluating the safety and tolerability of Sepofarsen in children under 8 with LCA…

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Diseases / indications

• Leber Congenital Amaurosis 10

Drugs mentioned

• Sepofarsen (sepofarsen)

Snippet

Sponsor: ProQR Therapeutics | Conditions: Leber Congenital Amaurosis 10, Blindness | Phase 2, Phase 3 | Status: UNKNOWN