Across America, millions of parents of children with rare diseases are in a race against time, hoping that new treatments will be developed fast enough to save their kids. Thanks to advances in ...

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 ...

NORD report shows 63 FDA vouchers driving breakthroughs for 47 rare pediatric diseases, 43 with no prior treatments—unprecedented progress for children left behind WASHINGTON, Dec. 4, 2025 /PRNewswire ...

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 ...