Morgan Ebert, Executive Editor

FDA accepted a levacetylleucine sNDA for ataxia-telangiectasia and set a September 19, 2026, target action date.

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.

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

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

Preclinical data for Gemma's GB703 in DMD and GB221 in SMA1 highlight early gene therapy strategies, but clinical questions remain.

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.

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.

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.

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

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.

New expert recommendations outline pediatric glucocorticoid dose reduction strategies after starting crinecerfont in classic CAH.

The review found no credible evidence linking HPV vaccination to serious adverse pregnancy outcomes or other long-term health risks.

Take a quick look at everything you may have missed in April, including the top FDA approvals and latest clinical updates.

FDA expands ASCENIV label to include pediatric patients aged ≥2 years with primary immunodeficiency, broadening earlier treatment access.

a2 Milk Company recalled 3 infant formula batches after detecting cereulide, a toxin that can cause rapid-onset gastrointestinal illness in infants.

FDA approved insulin glargine-aldy as an interchangeable biosimilar to Lantus for type 1 diabetes and adult type 2 diabetes.

FDA granted rare pediatric disease designation to investigational (Z)-endoxifen for McCune-Albright syndrome in girls.

Don Arnold, MD, MPH, highlights how a nurse-driven guideline reduced time to phototherapy in neonatal jaundice care.

Phase 3 data show berdazimer gel improved lesion clearance vs vehicle in molluscum contagiosum with a favorable safety profile.

A look back at the FDA submissions and regulatory decisions in the pediatric health care space from April 2026.