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

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

There’s a good chance that you haven’t heard about a rare condition called chromhidrosis. In a nutshell, the person afflicted with the disease produces colored sweat, from yellow to green, even red ...

Lozano is a rare disease mom, neuroscience Ph.D. candidate at UC Davis, and board member for the PURA Syndrome Foundation. In May, a historic moment in science and medicine was captured in a single ...

Families and researchers are advocating for Huntington's disease to be recognized as a rare disease in India to establish a ...

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

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 ...

This past spring, a biotech company announced the first use of a new gene-editing technology in people to fix an errant gene that causes a severe immune disorder. In June, a baby born with a ...

See more of our trusted coverage when you search. Prefer Newsweek on Google to see more of our trusted coverage when you search. When Casey McPherson became a father, his purpose became clear. He ...

An England-wide study of over 58 million people has identified eight rare diseases that carry significantly increased risks for COVID-19-related mortality in fully vaccinated individuals. This ...