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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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 a treatment for Friedreich ataxia after the results of randomized, placebo-controlled study indicate a strong effectiveness in treatment.
