genetic diseases

SEATTLE, Oct. 1, 2025 /PRNewswire/ — Despite a year marked by funding freezes, delays, and growing uncertainty about federal…

Hundreds of women have stepped up to help a Birmingham hospital detect rare genetic diseases. More than 1,200…

In Ionis Pharmaceuticals Inc.’s second U.S. FDA approval in under a year, the agency approved Dawnzera (donidalorsen) as…

(Yicai) Aug. 7 — Chinese researchers have developed two advanced DNA editing tools that could lead to breakthroughs…

BridgeBio Pharma, Inc. – By examining over 100 unique variants associated with the CASR gene causative of ADH1,…

Eight healthy babies have been born in the U.K. using a new IVF technique that successfully reduced their…

Pacific Northwest Research Institute Publishes Key Advances in Rare and Genetic Disease Research Amid National Funding Uncertainty