The FDA has approved a novel therapy for patients with hemophilia A or B without inhibitors. Clinical trials showed it significantly reduces bleeding episodes by targeting a new protein in the blood...
In a long-term follow-up to SIERRA, a multicenter, randomized, controlled phase 3 study, researchers found that patients 55 years of age or older with active relapsed or refractory acute myeloid leukemia...
In this retrospective study, researchers assessed the turnaround time of mutational tests in patients with newly diagnosed acute myeloid leukemia (AML) and the time from diagnosis to treatment initiation....
Researchers examined the epidemiology of hydroxymethylbilane synthase (HMBS) gene defects and activity levels among patients with acute intermittent porphyria to better understand the role of HMBS activity...
Individuals with acute hepatic porphyria often experience acute attacks and chronic symptoms which greatly diminish their quality of life. Researchers examined the efficacy of givosiran in reducing these...
Researchers examined if venetoclax—a therapy commonly used in the treatment of acute myelogenous leukemia and chronic lymphocytic leukemia—was associated with an increased risk of opportunistic infections,...
The FDA approved the first treatment for patients with congenital thrombotic thrombocytopenic purpura (cTTP), which is a rare and life-threatening blood clotting disorder that may be fatal if left...
Researchers set out to compare the time until the next treatment in patients with chronic lymphocytic leukemia who are treated with single-agent ibrutinib at a sustained dose of 420 mg/day vs patients who...
A recent phase 3 trial showed that for untreated patients with chronic lymphocytic leukemia, measurable residual disease-directed ibrutinib–venetoclax therapy had superior progression-free survival compared...
