LEIGH SYNDROME

Clinical trials for LEIGH SYNDROME explained in plain language.

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Not yet finished but already full! 1 Terminated 1

New hope for devastating childhood disease in drug trial

Disease control ENROLLING_BY_INVITATION

This study is testing whether a drug called sirolimus is safe and can help control symptoms in people with Leigh syndrome, a severe genetic disorder that affects the brain and muscles. About 15 participants, from infants to adults, will take the drug daily for up to two years. Re…

Matched conditions: LEIGH SYNDROME

Phase: PHASE2 • Sponsor: Matthew Demczko • Aim: Disease control

Last updated Apr 04, 2026 02:50 UTC

LEIGH SYNDROME

Get alerted when new LEIGH SYNDROME trials appear

New hope for devastating childhood disease in drug trial