One-Shot gene therapy tested in toddlers with devastating muscle disease
Disease control
Terminated
This study tested a one-time gene therapy injection for children aged 6 months to 5 years with a specific type of spinal muscular atrophy (SMA). The main goal was to find a safe dose and see if the treatment could help children gain motor skills, like standing or walking. The tri…
Phase: PHASE1 • Sponsor: Novartis Gene Therapies • Aim: Disease control
Last updated Mar 27, 2026 12:38 UTC