Pediatric Neurology

Myrtelle Inc. has reported promising 6-month trial data for a recombinant adeno-associated virus vector-based investigational gene therapy for Canavan disease.

Treatment with pitolisant demonstrated significant efficacy in reducing excessive daytime sleepiness and cataplexy in children aged 6 years and older, according to research presented in The Lancet Neurology.

Delandistrogene moxeparvovec improved motor function and was well-tolerated 4 years after treatment in a small cohort of children with Duchenne muscular dystrophy, according to a poster at the 2023 MDA Clinical & Scientific Conference.

Treatment with casimersen increased exon 45 skipping from baseline and was well-tolerated in children with Duchenne muscular dystrophy, according to a poster at the 2023 MDA Clinical & Scientific Conference.

Treatment with givinostat was associated with improvement in mobility and physical function compared with placebo in ambulant boys with Duchenne muscular dystrophy, according to a poster at the 2023 MDA Clinical & Scientific Conference.

The FDA has approved Lamzede, an enzyme replacement therapy for non-central nervous system manifestations of alpha-mannosidosis in adult and pediatric patients, Chiesi Global Rare Diseases announced in a press release.

In children with familial high risk of schizophrenia or bipolar disorder, evidence of motor impairment skills may indicate neurodevelopmental vulnerability for psychosis.

Neurogene Inc. announced the FDA has granted clearance to its investigational new drug application for NGN-401, an adeno-associated virus gene therapy candidate for treating Rett syndrome in female pediatric patients.

Myrtelle Inc. announced updated findings of the open-label phase 1/2 clinical trial of its recombinant adeno-associated virus vector-based investigational gene therapy for Canavan disease, a fatal genetic disorder in children.

Jessica Clymer, MD, has been appointed director of pediatric neuro-oncology at Hassenfeld Children’s Hospital at NYU Langone.