Pediatric Neurology News

The FDA has approved an oral, small molecule MEK inhibitor for adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas not amenable to complete resection.

The FDA has approved Evrysdi in tablet form for patients with spinal muscular atrophy aged 2 years and older who weigh more than 44 pounds, according to the manufacturer.

As the body of research expands on the potential of glucagon-like peptide-1 receptor agonists to address various health issues, recent data suggest they may improve brain health.

A Boston-area biopharmaceutical firm has submitted a biologics license application to the FDA for its investigational, fully human monoclonal antibody to treat individuals with spinal muscular atrophy.

The odds for stroke in older adults was significantly higher for those who experienced parental divorce by their 18th birthday compared to those who did not, and higher for those of an older age, data show.

The prevalence of intraventricular hemorrhage among preterm infants has not changed over time, but it occurs later in life than it did 20 to 30 years ago, according to the results of a large study published in JAMA Pediatrics.

A clinical-stage biotechnology company will advance a novel autologous chimeric antigen receptor T-cell therapy that targets the immune checkpoint B7-H3 for diffuse intrinsic pontine glioma, or DIPG, a rare incurable pediatric brain tumor.

LOS ANGELES — A wearable, behind-the-ear EEG device is feasible for assessment of sleep patterns in young people with Dravet syndrome, according to a poster presented at the American Epilepsy Society annual meeting.

Topline results from a phase 3 clinical trial showed an investigational gene therapy for young people with spinal muscular atrophy type 2 increased mobility and slowed disease progression, according to a press release from Novartis.