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

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 ...

The MarketWatch News Department was not involved in the creation of this content. -- Key presentations include two updates from a phase 3 trial evaluating investigational treatment with Mim8 ...

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

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.

“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 ...

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 ...

Joey Carlsen Martinez was treated at MSK for Erdheim-Chester disease, a rare form of blood cancer. He received an experimental drug called ulixertinib as part of a single-patient clinical trial. The ...

PLAINVILLE, Conn. (WFSB) - A local community is stepping up to help a young girl from Plainville with a rare blood disease. In Southington, there was a special blood drive to support 13-year-old ...

A newly identified and rare genetic variant slows the growth of mutated blood stem cells and reduces the risk of leukemia.

A former Campbell County Sheriff is now in remission after battling cancer. Terry Gaddy was diagnosed with a rare form of ...