BACKGROUND Cannabidiol has been used for treatment-resistant seizures in patients with severe early-onset epilepsy. We investigated the efficacy and safety of cannabidiol added to a regimen of conventional antiepileptic medication to treat drop seizures in patients with the Lennox–Gastaut syndrome, a severe developmental epileptic encephalopathy.  

Findings from a randomized, double-blind, phase 3 efficacy and safety trial published in the New England Journal of Medicine suggest that infants with spinal muscular atrophy who receive nusinersen experience greater survival and motor function improvement compared with controls.