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 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.