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 ...

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.

A single, untargeted proteomics test for rare genetic diseases has been developed. A research team from the University of Melbourne (Australia) and Murdoch Children’s Research Institute (Victoria, ...

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

Novo Nordisk is paying $240 million for global rights to a Phase 3-ready drug from Omeros that could rival immune disorder medications currently available from companies such as AstraZeneca, Novartis, ...

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 ...

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 ...

NORTH CHARLESTON, S.C. (WCSC) — A North Charleston child battling a rare disease is about to take the trip of a lifetime after spending nearly two years in the hospital going through treatment. Jaxson ...

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