FDA okays AQVESME as first treatment for anemia in alpha- & beta-thalassemia
The approval makes AQVESME the only FDA-approved therapy for anemia in both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia
The approval makes AQVESME the only FDA-approved therapy for anemia in both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia
Study published in laboratory investigation, the official journal of the United States and Canadian Academy of Pathology
The study found that nerandomilast effectively slowed lung function decline in patients, with discontinuation rates similar to placebo
Adverse events were consistent with valbenazine’s established safety profile
Finerenone is the first therapy targeting the mineralocorticoid receptor pathway to demonstrate cardiovascular benefits in heart failure patients
This Breakthrough Therapy Designation highlights the impressive clinical benefit of Enhertu over the current standard of care
Full approval will depend on verification of clinical benefit in a confirmatory trial
FOP is a rare genetic disorder caused by mutations in the ALK2 kinase
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