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

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.

THE BRONX, N.Y. (PIX11) — A 16-year-old with a rare blood disease was cured with innovative gene therapy by doctors at the Children’s Hospital at Montefiore. Miriya Nurse got emotional at the thought ...

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 ...

If your child were critically ill, how far would you go to find answers?

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

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, ...

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 ...

A Phoenix grandfather battling a rare blood disorder found a matching stem cell donor overseas, but his medical aid will not cover the necessary transportation expenses.

At 11 years old, Mykela Shields has experienced more hospital stays, tests and procedures than most people will in their lifetime. Her memories consist of the pinch of a blood draw, the ...