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.

Phase 3 CROSSING trial data show tezepelumab improved histologic remission and dysphagia versus placebo through week 52 in EoE.

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.

XOCOVA gives pediatricians a new oral option to help prevent COVID-19 after exposure in adolescents aged 12 years and older.

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

Phase 3 CANOPY-HCH-3 data show vosoritide significantly improved growth velocity, height, and arm span in children with hypochondroplasia.