The FDA approved tiratricol (Emcitate) for peripheral thyrotoxicosis in MCT8 deficiency, the first approved therapy for the rare disorder.

FDA approves oral ALK2 inhibitor zilurgisertib for FOP in patients 12+, based on phase 2 PROGRESS data on new heterotopic ossification volume.

Lavengratinib increased annualized height velocity in 7 children with achondroplasia, but open-label phase 2 findings remain preliminary.

The FDA accepted the dersimelagon NDA with priority review for EPP and XLP; a decision on the oral MC1R agonist is expected by February 2027.

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).

FDA approved apitegromab-mstn (Isembyld) for SMA in patients 2 years and up, based on phase 3 data showing added motor gains with SMN2 therapy.

Week 52 data from the reACHin trial show navepegritide stabilized foramen magnum stenosis and boosted growth in infants with achondroplasia.

At AAP 2026, Peter Lio, MD, discussed distinguishing allergic and inflammatory skin conditions and when pediatricians should seek specialty care.