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

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

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

Thousands of times per year, a family’s moment of joy turns to unexpected grief. A seemingly healthy infant stops smiling or ...

Jeff and Deena Leider are pushing federal officials to expand access to a treatment for their two sons' rare disease.

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

Rare diseases are defined as conditions affecting fewer than 200,000 people in the US or less than 1 in 2,000 in Europe. 1-2 While each disease is individually rare, collectively, they represent a ...

The Cyprus Institute of Neurology and Genetics (CING) will host a European conference on rare diseases on June 16, jointly organised with the Deputy Ministry of Research, Innovation and Digital Policy ...

More than 500 European patients with previously undiagnosed rare disorders received a genetic diagnosis through research carried out by an extensive European collaboration, the Solve-Rare Diseases ...

Rare diseases - a medical condition so obscure that even your doctor has to Google. Despite their name, these diseases collectively impact millions of Americans, creating a healthcare system that’s ...

Connecticut prides itself on strong schools, world-class healthcare, and a commitment to caring for its most vulnerable residents. We are a state that values education, innovation, and community. Yet ...