Contemporary Pediatrics week in review: September 14-18, 2026

Review some of the top stories from the Contemporary Pediatrics website over the last week, and catch up on anything you may have missed.

Thank you for visiting the Contemporary Pediatrics® website. Take a look at some of our top stories from last week (Monday, September 14 to Friday, September 18, 2026) and click each link to read and watch anything you may have missed.

1.) FDA approves rebisufligene etisparvovec, first gene therapy for Sanfilippo syndrome type A

The FDA approved Fayuvi (rebisufligene etisparvovec-hopf) as the first treatment for Sanfilippo syndrome type A (MPS IIIA), a rare and fatal pediatric neurodegenerative disorder that affects roughly 1.6 per 100,000 live births and, until now, could only be managed with supportive care; affected children typically die around age 15. The approval, from developer Ultragenyx Pharmaceutical, was based on an open-label, single-arm trial in children ages 2 to 5 with a median follow-up of 4.8 years (extending to 8.5 years), during which treated children maintained or improved cognitive function compared with the disease's expected natural decline. Adverse reactions occurring in more than 5% of patients included elevated liver enzymes, nausea, vomiting, fever, and cytopenias.

Because the trial relied on a historical comparison rather than a concurrent control group, the design leaves room for confounding, and researchers note that long-term oncogenic risk from the therapy's genomic integration remains theoretical and unproven over longer follow-up.

2.) FDA approves apitegromab-mstn as first muscle-targeted therapy for spinal muscular atrophy

The FDA approved apitegromab-mstn (Isembyld) on September 11 as the first muscle-directed therapy for spinal muscular atrophy (SMA), intended for use alongside existing SMN2-targeted treatments such as nusinersen or risdiplam rather than as a replacement, according to developer Scholar Rock. In the phase 3 SAPPHIRE trial of 188 nonambulatory patients ages 2 to 21, apitegromab improved Hammersmith Functional Motor Scale Expanded scores by 2.2 points over placebo, and 34.2% of treated patients gained at least 3 points on the scale versus 13.5% of those on placebo. Common adverse effects included upper respiratory infections, vomiting, cough, and an increased risk of fractures.

The primary efficacy analysis included only 156 of the 188 enrolled participants (those ages 2 to 12), and the drug has been studied only as an add-on to existing SMN2-targeted therapy, not as a standalone treatment.

3.) Updated AAP guidance changes iron deficiency screening and treatment in children

The American Academy of Pediatrics issued updated clinical guidance on preventing, screening for, and treating iron deficiency and iron deficiency anemia in children. The revised recommendations call for screening breastfed infants at 9 to 12 months and formula-fed infants at 15 to 18 months, with iron supplementation of 1 mg/kg/day starting by 4 to 6 months for breastfed infants. Treatment dosing is set at 3 mg/kg daily for young children and 65 mg daily for adolescents, using ferritin thresholds of below 20 ng/mL for children and below 30 ng/mL for adolescents; clinicians can expect hemoglobin to rise about 2 g/dL within a month of starting treatment for severe anemia.

The guidance notes that ferritin is an acute-phase reactant that can be falsely elevated in children with concurrent inflammatory conditions, which can complicate diagnosis in some patients.

4.) Atopic dermatitis linked to higher ADHD risk in children, with sleep disturbance emerging as key factor

A narrative review found that children with atopic dermatitis face a 30% to 50% higher risk of ADHD than the general pediatric population, with meta-analyses reporting odds ratios of 1.34 and 1.28 for the association. ADHD prevalence reached 15% to 16% among children with moderate to severe atopic dermatitis, compared with about 7% among those with mild disease, and children who had both atopic dermatitis and sleep disturbances had more than double (2.43 times) the odds of ADHD symptoms. A Mendelian randomization analysis, however, found no direct genetic causal link between the two conditions (odds ratio, 1.02), suggesting the relationship may stem from disease burden rather than shared genetics.

The review's authors caution that most of the epidemiologic evidence is observational, and much of the proposed inflammation-itch-neurobehavioral mechanism linking the two conditions is drawn from animal or adult studies rather than pediatric research, so it remains a hypothesis awaiting prospective testing in children.

5.) Tezepelumab meets co-primary and secondary endpoints in phase 3 eosinophilic esophagitis trial

Tezepelumab-ekko (Tezspire), an anti-TSLP monoclonal antibody from Amgen and AstraZeneca, met its co-primary and secondary endpoints in the phase 3 CROSSING trial for eosinophilic esophagitis (EoE), a condition that now affects more than 470,000 people in the US after a fivefold rise in prevalence since 2009. In the 368-patient trial of participants ages 12 to 80, the drug — administered subcutaneously every 4 weeks — improved esophageal histology and dysphagia scores at week 24 compared with placebo, with benefits sustained through week 52; nearly half of EoE patients don't achieve adequate control with current first-line therapies.

Amgen and AstraZeneca have not yet disclosed detailed efficacy figures, with full data expected to be presented at an upcoming medical conference, and specific safety data beyond a "generally consistent" safety profile were not provided.