Novartis NVS reported promising new safety and efficacy results from its phase III studies of investigational intrathecal onasemnogene abeparvovec (OAV101 IT) in a broad group of patients aged 2 to under 18 years with spinal muscular atrophy (SMA). OAV101 IT is an investigational, one-time gene therapy for patients with SMA. In the registrational phase III STEER study, OAV101 IT treatment resulted in a statistically significant 2.39-point…