Novartis Gene Therapies

This study tested a one-time gene therapy (AVXS-101) in 22 infants under 6 months old with spinal muscular atrophy type 1, a severe muscle-weakening disease. The goal was to see if the treatment helped babies sit independently for at least 30 seconds and survive without needing p…

This study tested a single dose of a gene therapy called AVXS-101 in 30 infants with spinal muscular atrophy (SMA) who were diagnosed before symptoms started. The goal was to see if the treatment could help them reach motor milestones like sitting and standing alone. Results show…

This study tested a one-time gene replacement therapy for infants under 6 months old with spinal muscular atrophy type 1, a severe genetic muscle-wasting disease. Researchers gave a single intravenous infusion of the therapy called onasemnogene abeparvovec-xioi to see if it could…

This study tested a one-time gene therapy for babies under 6 months old with spinal muscular atrophy type 1, a severe genetic disease that weakens muscles. The therapy aimed to deliver a working copy of a missing gene through a single intravenous infusion. Researchers measured wh…