WASHINGTON — The Food and Drug Administration has approved the first drug treatment for pediatric patients 12 and older with a rare blood disorder. The treatment for acquired thrombotic ...

A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the burden of lifelong transfusions. A single one-time gene therapy could free ...

A multicenter research effort yielded findings that may allow for more effective and personalized treatment of an underrecognized but serious blood disorder.Extended follow-up of individuals with ...

A new stem cell transplant technique that does not require the use of high-dose chemotherapy and radiation successfully cured an adult patient with congenital dyserythropoietic anemia, marking the ...

PharmaEssentia is looking to expand the reach of its FDA-approved rare blood cancer treatment Besremi to another rare blood disorder after reviewing encouraging results from a phase 3 study. In the ...

Blood clotting disorders can be inherited or acquired and lead to either excessive bleeding or too much clotting.

Researchers have made a groundbreaking discovery in the field of hematology, providing an explanation for spontaneous and unusual blood-clotting that continues to occur despite treatment with ...