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…
Phase: PHASE2 • Sponsor: Matthew Demczko • Aim: Disease control
Last updated Apr 04, 2026 04:20 UTC