The FDA expanded approval of exagamglogene autotemcel (Casgevy) for patients aged 2 years and older with sickle cell disease and recurrent vaso-occlusive crises or transfusion-dependent β-thalassemia.
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In a phase 3 RCT of children aged 3 to 17 years with achondroplasia, once-daily oral infigratinib significantly increased annualized height velocity vs placebo at 52 weeks.
A phase 3 randomized trial evaluated the efficacy and safety of fremanezumab for preventive treatment of episodic migraine in children and adolescents, addressing a significant evidence gap in pediatric...
The FDA approval expands the use of marstacimab-hncq (Hympavzi; Pfizer Inc.) for routine prophylaxis to prevent or reduce bleeding episodes in additional pediatric and inhibitor-positive hemophilia A and B...
Recent studies in children and adults associate long COVID with worsened academic performance, attention difficulties, subjective cognitive impairment, and persistent neurologic and psychiatric symptoms.
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March 2026 included several FDA drug and biologic approvals across a range of therapeutic areas. This timeline highlights the key alerts our publication covered throughout the month, giving readers a quick,...
A multisite surveillance study published in Pediatrics assessed trends and determinants of influenza antiviral use in hospitalized children across the United States before and during the late COVID-19...
The FDA has approved Avlayah (tividenofusp alfa-eknm) for certain pediatric patients with Hunter syndrome, marking the first approval targeting the disease's neurologic complications and offering clinicians...
