Most of the rare diseases are inherited—passed down from parents to their children. Generally referred to as orphan diseases, each of the almost 7,000 of them is due to the same issue—a mutation in a ...

For families with children facing rare medical conditions, hearing that treatment will require neurosurgery is an immensely overwhelming moment. Instantly, deep fear sets in and critical questions ...

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 ...

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 ...

A new study shows children and young people face long-lasting and higher risks of rare heart and inflammatory complications after COVID-19 infection, compared to before or without an infection.

"America's rare children cannot afford another month of inaction." "Lennox-Gastaut Syndrome (LGS) is a devastating childhood epilepsy, and for years, families had no options. The PRV program finally ...