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

The lifelong effects of rare blood disorder diagnoses in childhood are being analysed in a first-of-its-kind study led by 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 ...

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 ...

Swedish Orphan Biovitrum (BIOVF) (SWOBY), the Nordic pharma company also known as Sobi, announced on Friday that the European ...

Major new analysis from the Thrombotic Thrombocytopenia Syndrome (TTS) Consortium has provided critical insights into the rare but serious condition known as vaccine-induced immune thrombotic ...

“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 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, ...

A new, rapid testing method will greatly help the diagnosis of rare diseases in babies and children, according to research to be presented to the annual conference of the European Society of Human ...

NORWELL, Mass., Dec. 8, 2025 /PRNewswire/ -- The National Organization for Rare Disorders (NORD®), a leading rare disease patient advocacy organization, today announced the appointment of Kim Isenberg ...

Scientists have discovered the rarest blood type on Earth, belonging to just one woman. Talk about a lonely hearts club. An unidentified woman from Guadeloupe has the rarest blood type on Earth, to ...