Only four people in Australia have been diagnosed with rare genetic disease CHOPS
Isla Steed is your fairly typical teenager, according to her mum. The 14-year-old is “a stubborn little person”…
Pacific Northwest Research Institute Publishes Key Advances in Rare and Genetic Disease Research Amid National Funding Uncertainty
SEATTLE, Oct. 1, 2025 /PRNewswire/ — Despite a year marked by funding freezes, delays, and growing uncertainty about federal…
More than 1,000 women sign up for vital Birmingham study to detect rare genetic diseases
Hundreds of women have stepped up to help a Birmingham hospital detect rare genetic diseases. More than 1,200…
Ionis gets approval for preventing attacks in rare and genetic HAE
In Ionis Pharmaceuticals Inc.’s second U.S. FDA approval in under a year, the agency approved Dawnzera (donidalorsen) as…