First Oral SMA Treatment Gets EMA Panel Thumbs Up

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended marketing authorization for risdiplam (Evrysdi), the first treatment that can be given orally to patients with certain types of spinal muscular atrophy (SMA). Risdiplam is “expected to increase survival of motor neurons and reduce symptoms of the disease,” […]