Could a common drug help kids with rare leigh syndrome?
Disease control
ENROLLING_BY_INVITATION
This study tests the drug sirolimus (rapamycin) in 15 people aged 6 months to 55 years with genetically confirmed Leigh syndrome, a rare and serious mitochondrial disease. The goal is to see if the drug is safe and can help with symptoms like muscle weakness and developmental del…
Phase: PHASE2 • Sponsor: Matthew Demczko • Aim: Disease control
Last updated Jun 27, 2026 12:01 UTC