The treatment for acquired thrombotic thrombocytopenic purpura, a rare blood disorder that causes blood clots in the small ...

The U.S. Food and Drug Administration declined to approve Atara Biotherapeutics' cell therapy for a rare form of blood cancer ...

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 team of Australian researchers have developed a new targeted therapeutic approach that could improve treatment for ...

A New York City teen was cured of a rare blood disease — just in time to spend the holidays at home with her family — thanks to a breakthrough treatment. Miriya Nurse, 16, was first diagnosed with ...

The human body is always coming up with new ways to shock and surprise us, and it turns out that just 50 known people in the ...

“This partnership marks a major milestone in our mission to transform how rare disease therapies reach patients in Europe,” said Adam Plich, Founder and CEO of Avanzanite. “We provide biotech partners ...

A new "proteomic" blood test can accurately screen a newborn's DNA for thousands of rare, inherited diseases by analyzing just a single-milliliter sample, according to a study to be presented Monday.

In a major leap for genetic diagnostics, scientists from the University of Melbourne and the Murdoch Children's Research Institute (MCRI) have developed a groundbreaking rapid blood test that can ...

Hutchmed is preparing to file for approval of a drug for the rare autoimmune disorder warm antibody autoimmune haemolytic ...