Abstract Objective: To compare the efficacy and safety of intravenous Levetiracetam and Phenobarbitone in the treatment of neonatal seizures. Design: Open labelled, Randomized controlled trial. Setting: Level III Neonatal Intensive Care Unit (NICU). Participants: 100 neonates (0-28 days) with clinical seizures. Intervention: If seizures persisted even after correction of hypoglycemia and hypocalcemia, participants were randomized to receive either Levetiracetam (20 mg/kg) or Phenobarbitone (20 mg/kg) intravenously. The dose of same drug was repeated if seizures persisted (20 mg/kg of Levetiracetam or 10 mg/kg of Phenobarbitone) and changeover to other drug occurred if the seizures persisted even...

Background Phenytoin is the recommended second-line intravenous anticonvulsant for treatment of paediatric convulsive status epilepticus in the UK; however, some evidence suggests that levetiracetam could be an effective and safer alternative. This trial compared the efficacy and safety of phenytoin and levetiracetam for second-line management of paediatric convulsive status epilepticus.

Paolo Curatolo, David N Franz, John A Lawson, Zuhal Yapici, Hiroko Ikeda, Tilman Polster, Rima Nabbout, Petrus J de Vries, Dennis J Dlugos, Jenna Fan, Antonia Ridolfi, Diana Pelov, Maurizio Voi, Jacqueline A French https://doi.org/10.1016/S2352-4642(18)30099-3 Background Epilepsy occurs in 70–90% of patients with tuberous sclerosis complex. We aimed to assess the efficacy and safety of adjunctive everolimus for treatment-refractory seizures associated with tuberous sclerosis complex in paediatric patients enrolled in the EXIST-3 trial, a double-blind, placebo-controlled, randomised, phase 3 study. Methods This post-hoc analysis focused on paediatric patients...

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.

Background Duchenne muscular dystrophy (DMD) is a severe, progressive, and rare neuromuscular, X-linked recessive disease. Dystrophin deficiency is the underlying cause of disease; therefore, mutation-specific therapies aimed at restoring dystrophin protein production are being explored. We aimed to assess the efficacy and safety of ataluren in ambulatory boys with nonsense mutation DMD.