Scientists have uncovered the precise molecular trigger behind a rare blood-clotting disorder linked to certain COVID-19 ...

Medical case study: doctors navigated complex risks and used a tailored treatment strategy to successfully transplant a kidney from the patient's elder brother.

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 ...

This year’s Rare Disease Day event Feb. 27 at the Jacobs School of Medicine and Biomedical Sciences focused on updates in research and diagnostics, and advancements in treatment.

Ultragenyx Pharmaceutical Inc. RARE shares are down on Thursday as the company reported data from its Phase 3 study of DTX301 ...

Artificial intelligence is rapidly inventing new drugs for diseases from Parkinson's disease and antibiotic-resistant superbugs to rare lung conditions.

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 ...

Odisha's health department has introduced two advanced medicines for haemophilia and Von Willebrand disease patients, marking a first for the state. These extended half-life Factor VIII and Von ...