The National Institutes of Health has thrown its hat into the drug development ring with the announcement of a new program that will seek to bring drugs that treat rare and neglected diseases onto the ...

Jim Higgins has an inherited cancer syndrome that has killed his father, sister, and two daughters. He fears NIH cuts will ...

FDA Commissioner Marty Makary’s proposal to roll out a new drug approval pathway is heightening hope for the rare disease ...

The National Institutes of Health has withdrawn renewal grant applications for the Consortium of Eosinophilic Gastrointestinal Disease Researchers. Patient advocacy organizations, including the ...

For the past 20 years, Janet Edmunson has run support groups for patients with rare neurological diseases that have no ... the National Institutes of Health (NIH) under the Trump administration ...

An HHS official told USA TODAY that the department is not creating an autism registry, but is pursuing the real-world data ...

according to the National Institutes of Health (NIH). However, only 5% of rare diseases are estimated to have at least one approved treatment, known as “orphan” therapies. A rare disease is ...

How Worldwide Clinical Trials’ Rally for Rare Event Came About One of the most influential events in rare disease is the FDA–NIH Rare Disease Day conference, which allows the rare disease community to ...