Gene therapy trial targets kids with spinal muscular atrophy who can sit but not stand
Disease control
Terminated
This study tested a gene therapy called AVXS-101 in children aged 6 months to 5 years with spinal muscular atrophy (SMA) who could sit but not stand or walk. The goal was to see if the treatment was safe and could help them gain motor skills like standing or walking. The trial wa…
Phase: PHASE1 • Sponsor: Novartis Gene Therapies • Aim: Disease control
Last updated May 15, 2026 11:54 UTC