Experimental gene therapy offers hope for children with rare brain disease
Disease control
ENROLLING_BY_INVITATION
This study tests a one-time gene therapy, called AVASPA, for children with Canavan disease, a rare and severe brain disorder. The therapy is given directly into the brain during a surgical procedure. The goal is to restore the missing enzyme and improve brain function. The trial …
Phase: PHASE1, PHASE2 • Sponsor: Myrtelle Inc. • Aim: Disease control
Last updated May 14, 2026 12:04 UTC