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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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...
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...
The FDA has approved concizumab-mtci, a daily subcutaneous treatment to reduce bleeding for patients with hemophilia who have clotting factor inhibitors.
The FDA has approved a treatment for Friedreich ataxia after the results of randomized, placebo-controlled study indicate a strong effectiveness in treatment.
