Chapter 5: How to prescribe neffy

FDA approved levacetylleucine (Aqneursa) for ataxia in ataxia-telangiectasia, the first therapy for the disease, in patients 15 kg or heavier.

Tallie Z. Baram, MD, talks about how adding a measure of childhood unpredictability to ACEs screening may help pediatricians identify children at risk for mental health problems.

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

The FDA granted priority review to efzimfotase alfa for HPP in patients aged 2 years and older; a decision is expected in the first half of 2027.

Adolescent VALIANT subgroup data show pegcetacoplan cut proteinuria 75% vs placebo, supporting its expanded pediatric kidney indication.

The FDA approved Fayuvi (rebisufligene etisparvovec-hopf), a one-time AAV9 gene therapy, as the first treatment for pediatric MPS IIIA (Sanfilippo syndrome type A).

Aquestive resubmitted its NDA for dibutepinephrine sublingual film Anaphylm, citing new human factors and PK data addressing the FDA's CRL.

Melissa Wallach, MD, FAAP, discusses what pediatricians should know about updated screening, supplementation, and treatment recommendations for iron deficiency.