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

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 ...

Pursuing Elusive Diagnoses for Rare Diseases Dr William Gahl describes an NIH program designed to help patients and healthcare providers who have spent years seeking diagnoses for chronic ...

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 ...

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 ...

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

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 ...

An FDA quality control program for testing milk and other dairy ... spoke about an expedited rare disease approvals pathway. (BioSpace) ...