One-time gene replacement therapy improves motor function in SMA patients
04 Jun 2026
byAudrey Abella
Intrathecal onasemnogene abeparvovec (OAV101 IT), a one-time gene transfer therapy, significantly improves motor function in treatment-naïve patients with spinal muscular atrophy (SMA), as shown by the period 1 results of the phase III STEER study.