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New method brings growth charts to children with rare genetic condition

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 ...
STAT

Parents of children with rare diseases ask: How long until our CRISPR miracle?

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 ...
Morningstar

America's Rare Children Need Congress to Act: NORD Urges Swift Reauthorization of Proven Rare Pediatric Disease Voucher Program

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 ...
KWTX

Parents keep faith as their children battle a rare disease, affecting only 30 kids in US

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 ...