Pediatric safety and pharmacokinetic findings supported expanding leniolisib to children aged 4 to 11 years who weigh at least 27 kg, establishing the first FDA-approved treatment for activated...
Clinical and pharmacokinetic evidence supported approval of zilganersen across pediatric and adult age groups, providing the first therapy designed to reduce production of the abnormal GFAP protein...
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.
In a landmark survival analysis of 596 consecutive patients with transthyretin amyloid cardiomyopathy, worsening NT-proBNP and heart failure hospitalization at 6 months were independently associated with...
A post-hoc analysis presented at the American College of Cariology’s Annual Scientific Session suggests that maintaining functional stability, measured by 6-minute walk distance, is associated with lower...
A new multicenter analysis presented at the 67th American Society of Hematology (ASH) Annual Meeting evaluates how often emergency departments meet national guidelines for timely opioid administration in...
A phase 3 clinical trial demonstrates the efficacy of olezarsen in reducing plasma triglycerides and acute pancreatitis episodes in patients with familial chylomicronemia syndrome.
