BLINDNESS

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Trials to join now! 1 Not yet recruiting 1 Not yet finished but already full! 1

Experimental drug aims to restore sight in rare childhood blindness

Disease control Recruiting now

This study tests an investigational drug called sepofarsen in people with Leber congenital amaurosis (LCA), a rare genetic eye disease that causes severe vision loss from birth. The trial includes 32 participants aged 6 and older who have a specific mutation in the CEP290 gene. E…

Matched conditions: BLINDNESS

Phase: PHASE3 • Sponsor: Laboratoires Thea • Aim: Disease control

Last updated May 13, 2026 15:59 UTC

BLINDNESS

Get alerted when new BLINDNESS trials appear

Experimental drug aims to restore sight in rare childhood blindness