Could a common drug slow a rare brain disease?
Disease control
ENROLLING_BY_INVITATION
This study tests the drug sirolimus in 15 people with Leigh syndrome, a rare genetic disorder that damages the brain and muscles. The goal is to see if sirolimus is safe and can help manage symptoms. Participants must be between 6 months and 55 years old and have a confirmed gene…
Phase: PHASE2 • Sponsor: Matthew Demczko • Aim: Disease control
Last updated May 18, 2026 12:03 UTC