Pediatric Neurology News

Treatment with IV nipocalimab was safe and led to significant reductions in disease expression at 24 weeks in adolescents with generalized myasthenia gravis, according to data presented at AANEM.

An oral therapeutic significantly reduced the number of tumors in adults with neurofibromatosis type 1 who have symptomatic, inoperable plexiform neurofibromas, according to topline results from a phase 3 study.

Low-level lead poisoning remains prevalent across the United States and globally, warranting screening and identification of those at highest risk, according to a recently published review.

Pregabalin and topiramate significantly reduced the frequency and intensity of migraines in children and adolescents, especially when combined with vitamin supplements, according to a meta-analysis published in JAMA Network Open.

In pregnant women with MS given ocrelizumab, treatment did not result in infant B-cell depletion after birth, and the drug was virtually undetectable in the bloodstream of mother and child, according to a poster from ECTRIMS 2024.

Sleep disturbance at age 10 years was associated with a greater risk for suicidal ideation and behavior by age 12, underscoring the importance of sleep in the prevention of youth suicide, according to research published in JAMA Network Open.

The European Medicines Agency has granted orphan drug designation to a small molecule intended to treat spinal and bulbar muscular atrophy, according to the manufacturer.

Bluebird Bio’s lentiviral vector gene therapy Skysona met its primary efficacy endpoint of survival without major functional disability in 81% of boys with early cerebral adrenoleukodystrophy, a recent analysis showed.

Topline results from the phase 3 SAPPHIRE clinical trial showed that an investigational muscle-targeted therapy improved motor function in patients with spinal muscular atrophy compared with placebo as early as week 8.