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.
...
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...
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...
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.
Plozasiran, a first-in-class RNA interference therapeutic, reduced triglycerides by 80% and lowered the risk of acute pancreatitis by 83% in patients with familial chylomicronemia syndrome, earning FDA...
