Neurology

FDA approves Itvisma for patients 2 years and older with SMA, making it the first FDA-approved gene replacement therapy for this population.

The clearnace was granted to the Ceribell System for electrographic seizure detection in newborns pre-term and older.

"What is the possibility for your individual child that you're taking care of as a patient, as a family member advocate?" said Oliva Kim-McManus, MD.

FDA adds boxed warning to Elevidys and limits use to ambulatory DMD patients ≥4 years after reports of fatal acute liver failure in non-ambulatory patients.

New research shows an increase in neurodevelopmental disorder risk among children exposed to maternal COVID-19 infection in utero.

At CNS 2025, Kathryn Xixis, MD, highlighted the growing adoption of EPAs and outcomes-based learning in pediatric neurology.

At the 2025 CNS Annual Meeting, Craig Press, MD, PhD, discussed challenges in achieving equitable access to diagnostics and expertise in pediatric neurocritical care.

Craig Press, MD, PhD, discussed the evolution of pediatric neurocritical care toward individualized care at the 2025 Child Neurology Society meeting.

The BeeLine trial is a placebo-controlled study designed to evaluate radiprodil’s impact on both seizure frequency and non-seizure symptoms associated with GRIN-NDD.

Thomas Crawford, MD, said SAPPHIRE trial data show apitegromab provides clinically meaningful strength gains for children with SMA by targeting muscle in addition to motor neurons.

The FDA announces significant updates on acetaminophen risks in pregnancy and approves leucovorin calcium for treating CFD in children.

Zilganersen met the primary endpoint of gait stabilization, showing statistically significant benefit on gait speed and favorable safety.

According to new, real-world data for trofinetide, caregivers reported quality-of-life, alertness, and interaction improvements at 12 months of treatment.

Ji-Hyun Lee reports that short surgical exposure to sevoflurane did not affect IQ, behavior, or language outcomes in infants and toddlers.

Results suggest that a single, brief exposure to general anesthesia for surgery is unlikely to impair short-term neurodevelopment in young children.

If approved, vatiquinone would have been the first therapy approved for pediatric patients with FA.

Additionally, long sleep duration could be associated with increased odds of being reliably symptomatic at 4 weeks.

The patient described the headache as “pressure-like,” episodes that occurred for 1 to 2 hours with high severity.

Currently, omaveloxolone (Skyclarys) is approved to treat adults and adolescents aged 16 years and older impacted by FA.

In this article, we recap our top stories, expert interviews, and Q+A discussions from the 2025 Pediatric Academic Societies meeting.

New data from national survey highlights disparities and need for focused prevention strategies.

In addition, data presented at PAS revealed a predictable pharmacokinetic profile in the newborn population, consistent with adult humans.

Diazepam nasal spray (Valtoco) is approved for short-term treatment of seizure clusters, now in patients aged 2 years and older.

In the immediate skin-to-skin contact group, improved breastfeeding practices up to 12 months were observed.