AveXis SMA gene therapy advances toward pivotal trial – FierceBiotech
FierceBiotechAveXis SMA gene therapy advances toward pivotal trialFierceBiotechAveXis has posted top-line data from a phase 1 trial of its gene therapy against spinal muscular atrophy (SMA). All 15 patients were event-free at 13.6 months of age, a sign… Continue reading
