News

Early phase 2 data suggest zovegalisib may reduce lesion volume and improve symptoms in PIK3CA-driven vascular anomalies.

Early OTC-HOPE data link ECUR-506 to fewer hyperammonemic events in infants with neonatal-onset OTC deficiency.

BMN 401 met a biochemical end point but missed radiographic improvement in a phase 3 trial of children with ENPP1 deficiency.

Rohit P. Shenoi, MD, highlighted updated AAP drowning prevention guidance focused on supervision, swim skills, and equity.

New AAP guidance highlights layered drowning prevention strategies, including supervision, swim lessons, CPR training, and pool fencing.

After accounting for maternal mental health conditions, a new meta-analysis found no significant link between prenatal antidepressant exposure and autism or ADHD in children.

High school students who viewed tanning positively or sunscreen use negatively were less likely to practice sun safety

Hannah Murphy, PhD, of Vizient, discusses recent research that found children with diabetes had substantially higher rates of mental health disorders and suicide risk.

Researchers at Children’s Healthcare of Atlanta and Georgia Tech are testing an AI tool to detect worsening teen mental health online.

RGX-202 met a microdystrophin biomarker endpoint in phase 3 Duchenne muscular dystrophy data, with functional findings still preliminary.

A UK phase 2b trial will evaluate Lactin-V for preventing preterm birth in pregnant patients at high risk of early delivery.

National pediatric and infectious disease groups convened a measles summit as US cases reached the highest level in 35 years.

For Healthy and Safe Swimming Week, the CDC highlighted key strategies to prevent drowning, pool illnesses, and chemical injuries.

Expert recommendations outline how crinecerfont may help reduce glucocorticoid exposure in children with classic CAH.

Chronic pediatric skin disorders cause significant stigma, bullying, depression, and suicide risk—conditions often underrecognized and undertreated by physicians.

RVL-001, a vorinostat formulation, has entered early placebo-controlled studies in Rett syndrome and Pitt-Hopkins syndrome.

An international consensus group has recommended renaming PCOS to PMOS to better reflect the condition’s endocrine and metabolic features.

Expert recommendations outline how crinecerfont may help reduce glucocorticoid exposure in children with classic CAH.

Early AK-OTOF trial data showed hearing improvements and favorable safety in children with OTOF-mediated hearing loss.

Third-trimester maternal COVID-19 vaccination reduced infant hospitalization risk, while prepregnancy vaccination showed no protection.

The FDA approved ocrelizumab for pediatric RRMS in patients 10 years and older after positive OPERETTA II trial results.

ABS-1230 entered phase 1b/2 testing for KCNT1-related epilepsy as Actio also joined the FDA Rare Disease Evidence Principles process.

Researchers identified macrophage-driven dural lymphatic refinement as a potential regulator of social behavior in ASD models.

More than 20,000 water bead ingestions in children younger than 6 years were reported over 6 years, with some cases requiring surgery.

Urine F2-isoprostanes were associated with more severe viral respiratory illness in infants across 2 independent cohorts.

Modeling data suggest removing universal hepatitis B birth-dose vaccination could increase neonatal and chronic HBV infections in US infants.

Swedish trial data showed slow up-dosing peanut oral immunotherapy achieved sustained tolerance in most toddlers with peanut allergy.

Two-year data from the pivotal ApproaCH trial showed sustained growth improvements with TransCon CNP in pediatric achondroplasia.