Veterans fighting the muscle disease known as IBM say they want the U.S. government to recognize their condition as ...

Flesh turning to stone sounds like something from fantasy, but this rare genetic disease does something similar, often doing ...

CNBC Cures aims to help raise awareness of rare diseases and improve patient outcomes for people living with them. Led by "Squawk Box" anchor Becky Quick, the initiative was inspired by her family's ...

One in 5 of the highest-revenue drugs of 2022 was exclusively approved for rare conditions, accounting for more than 7% of US pharmaceutical spending. Study Design: Cross-sectional study. Methods: ...

Mississippi is launching a Rare Disease Task Force within the Mississippi Rare Disease Advisory Council to study issues ...

Students, clinicians, researchers and advocates will gather at UB on Feb. 27 for Rare Disease Day, an event that aims to raise awareness about rare diseases through education, collaboration and ...

Our inbox has been flooded with emails from viewers and readers who told us how their lives have been affected by rare diseases. Here are some of the stories we received. Living with a rare disease ...

Communication gaps, preventable delays and limited support place significant responsibility on patients on the rare disease journey, a new survey finds. The survey (PDF) was conducted by PANTHERx Rare ...

Many challenges face developers of drugs for rare diseases, including the small number of patients available for clinical trials, genotypic and phenotypic diversity within a disorder, poorly ...

As the grandfather of a girl with a rare disease, I’ve learned that for families like ours, hope often comes not as a cure, but as a clinical trial: carefully designed but inevitably exclusive. My ...

Ultragenyx’s Rare Bootcamp gives families a roadmap to help drive research and develop new treatments for rare diseases.