My niece Caitlin arrived at Boston Children’s Hospital unconscious, unresponsive, with a 105-degree fever. She needed ...
In an exclusive interview with PEOPLE, Jay and Pamela — both born with osteogenesis imperfecta, or brittle bone disease — ...
Sarepta Therapeutics Inc.'s stock tumbled 23% on Tuesday after the biotech said a patient who used its gene therapy Elevidys for the rare disease Duchenne muscular dystrophy has died of acute liver ...
Eight years after receiving a life-shattering diagnosis, a New Jersey mother credits an “amazing” new drug for stopping her ...
A Spanish hospital has achieved a rare feat — performing a sixth kidney transplant on a single patient. How did they do it, and what does this mean for the future?
ROCKY HILL, Conn. (WFSB) - First responders continue to meet the request for a young girl with a rare disease. Earlier this ...
Authorities confirm Betsy Arakawa died from a rare rodent-borne disease, days before Gene Hackman's death from heart disease.
The Pediatric Undiagnosed Diseases Program (UDP) at NYU Langone Health is a specialized program designed to find definitive ...
With rare diseases affecting 1 in 10 Americans and over 7,000 known conditions, the Cherokee Nation is taking a significant ...
Rare diseases affect one in 17 people in the UK, but a Government initiative designed to improve their quality of life will ...
The Cherokee Nation Health Services Rare Disease Task Force has released a report outlining progress made over the past year ...
With thousands of rare diseases affecting millions of families worldwide, one Tampa Bay lawmaker is taking action to position Florida as a national leader in genetic medicine.