Sanofi wins approval for first Bruton’s tyrosine kinase inhibitor in immune thrombocytopenia
1. The FDA approved rilzabrutinib, branded Wayrilz, as the first BTK (Bruton’s tyrosine kinase) inhibitor for adults with ITP (immune...
Hematology
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2 Minute Medicine: Pharma Roundup- Obesity RNA deal, at-home Alzheimer’s dosing, oncology royalty financing, and first Bruton’s Tyrosine Kinase Inhibitor for Immune Thrombocytopenia [September 3, 2025]
Novo’s srRNA push expands cardiometabolic pipeline On August 28, Novo Nordisk announced a collaboration with Replicate Bioscience to pursue self...
Revumenib may be safe and effective in the treatment of nucleophosmin 1-mutated acute myeloid leukemia
1. In pretreated patients with nucleophosmin 1-mutated (NPM1m) acute myeloid leukemia (AML), nearly half of patients treated with revumenib showed...
Artificial intelligence-guided ultrasound lacks sufficient accuracy for deep vein thrombosis detection
1. Curry and colleagues tested an artificial intelligence (AI) software that guided non-radiology specialists in diagnosing proximal deep vein thrombosis...
Mitapivat improves clinical outcomes in patients with non-transfusion dependent thalassemia
1. Hemoglobin response was significantly greater in the treatment group compared to placebo. 2. Majority of adverse events were mild...
Early anticoagulation after stroke decreases recurrence without increasing bleeding in patients with atrial fibrillation
1. Recurrent ischemic stroke rates were significantly reduced in patients who started DOAC within 4 days of an acute ischemic...
Red cell distribution width to albumin ratio may be a useful prognostic marker in the critical care setting
1. Higher red cell distribution width to albumin ratio (RAR) values were associated nonlinearly with a higher mortality risk. 2....
AAV gene therapy shows durable benefit in hemophilia B
1. In this prospective cohort study, a single intravenous dose of scAAV2/8-LP1-hFIXco gene therapy in men with severe hemophilia B...
Hereditary erythrocytosis is associated with a fetal-like EPO isoform
1. This case series identified three non-coding erythropoietin (EPO) variants that significantly increased gene expression under hypoxic conditions. 2. The...
Reduced-dose apixaban noninferior to full dose for cancer-associated thrombosis
1. Among patients with active cancer and prior venous thromboembolism (VTE), extended treatment with reduced-dose apixaban demonstrated noninferior efficacy to...
