Life-saving treatments for children with orphan illnesses often move slowly, if at all, through the federal approval process.

Every year, thousands of parents hear the heart-wrenching news that their child has been diagnosed with a rare disease, news that upends their world. Rare diseases do not discriminate, claiming ...

Growth charts for children with rare genetic disorders—giving health care professionals and families clearer guidance on how a child is developing—have been created by an international team, led by ...

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 ...

QUINCY, Ill. (WGEM/Gray News) - The Kempf family says they have received devastating news about their children when it comes to a rare disease. Both of their children have been diagnosed with ...