Instead of requiring personalized gene edits for each patient, the new approach could create a standardized method to use for ...

This is an archived article and the information in the article may be outdated. Please look at the time stamp on the story to see when it was last updated. NEW YORK (AP) — A germ that causes a rare ...

The National Institutes of Health established the Genomics Research to Elucidate the Genetics of Rare Diseases (GREGoR) Consortium in 2021 with the goal of finding molecular diagnoses for individuals ...

Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to ...

KAMPALA, (March 20, 2025) - A rare tropical disease that is the leading infectious cause of blindness worldwide was halved in one of the world’s worst-affected communities due to foreign aid but is ...

Scorpion sting envenomation meets all the criteria to be recognised as a neglected tropical disease, yet it remains ...

Uncommon illnesses caused by bacteria, viruses, fungi, or parasites fall under the category of rare infectious diseases. These illnesses are often caused by unique pathogens that affect very few ...

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 ...

The FDA has made some modest efforts, but it needs an internal organization that understands and would be an aggressive advocate for the development of rare-disease drugs. When I joined the FDA to be ...

Rare diseases reveal that our sense of identity rests on fragile biological infrastructure, much of it operating outside ...

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 ...

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 ...