Spinal muscular atrophy, SMA, a genetic neuromuscular disorder with progressive muscle wasting due to mutation in the SMN1 gene, deficiency in SMN protein, and loss of motor neurons, 3D illustration ...

The Phase III STEER study met its primary endpoint showing an increase from baseline in HFMSE total score in patients with SMA treated with intrathecal onasemnogene abeparvovec (OAV101 IT) OAV101 IT ...