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.

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

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 ...

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.

Researchers at the Sanford Stem Cell Institute have taken the fight against rare diseases to the lab, to patients’ bedsides ...

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 ...

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, ...

Disc Medicine is laying off a fifth of its workforce as the biotech reels from the FDA’s rejection of its rare blood disease candidate. | Disc Medicine is laying off a fifth of its workforce as the ...

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.

Fletcher Draper, a nine-year-old diagnosed with a rare blood disorder, celebrated his remission with a Disney-themed send-off at Landmark Credit Union Corp, thanks to the Make-A-Wish Foundation, which ...