Gene therapy breakthrough offers hope for rare brain disease
Disease control
ENROLLING_BY_INVITATION
This study tests a single dose of AVASPA gene therapy given directly into the brain of children with Canavan disease, a rare and severe genetic disorder that damages white matter. The therapy aims to restore a missing enzyme to help the brain produce myelin and slow disease progr…
Phase: PHASE1, PHASE2 • Sponsor: Myrtelle Inc. • Aim: Disease control
Last updated Jun 27, 2026 07:57 UTC