Guest Opinion. The path forward for rare disease patients and their families historically has been rocky, littered with ...

Primary sclerosing cholangitis, PSC in short, is a rare liver disease that destroys the bile ducts. It is a severe condition ...

Rare diseases affect an estimated 1 in 10 Americans and consist of over 7,000 known conditions. These staggering figures ...

Reuters, the news and media division of Thomson Reuters, is the world’s largest multimedia news provider, reaching billions ...

Health and Human Services Secretary Robert F. Kennedy Jr. said on Thursday that the U.S. drugs regulator would look for ways ...

In comments posted in response to the Trump administration’s pharma tariff investigation, companies and industry groups ...

A new agreement between the FDA and Avidity Biosciences could further assuage industry concerns about the agency’s stance on ...

The illness is caused by a parasite, which makes it different from many well-known tick-borne diseases, such as Lyme.

This is a developing news story. Please check back soon for updates. The FDA granted VG801 rare pediatric disease designation ...

Trump health officials signal eased FDA rules for rare disease gene therapy, but concerns linger over safety, oversight, and ...

For decades, researchers have wondered why some people with risky gene variants go on to develop Parkinson’s disease, while ...

Can a small fish help identify possible treatments for an ultra-rare inherited disease found in an Alabama boy? The genetic ...