Novartis Gene Therapies

This study tested a single-dose gene replacement therapy for babies under 6 months old with a severe form of spinal muscular atrophy (SMA Type 1). The main goal was to see if the treatment could help these infants achieve the milestone of sitting up on their own for at least 10 s…

This study tested a single-dose gene replacement therapy for infants under 6 months old with spinal muscular atrophy (SMA) type 1, a severe genetic disease that weakens muscles. The goal was to see if the one-time intravenous treatment could help babies achieve major milestones l…

This study tested a single-dose gene therapy for babies under 6 months old with a severe, life-threatening genetic muscle disease called Spinal Muscular Atrophy (SMA) Type 1. The goal was to see if the one-time intravenous (IV) treatment could help infants achieve major milestone…

This study tested a single, one-time gene therapy injection in infants who were diagnosed with spinal muscular atrophy (SMA) but had not yet shown symptoms. The goal was to see if delivering a working copy of a missing gene could prevent or control the severe muscle weakness and …