Scientists design new RNA therapy to bypass mutations behind thousands of genetic diseases
Scientists engineered transfer RNA to bypass premature genetic stop signals and restore production of full-length proteins in cystic…
Back on his feet: Thousands fund Israeli boy’s $3.2m degenerative disease treatment
Meitar Roz hopes to go skiing one day. Putting aside the scarcity of Israeli snow, it would not…
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…
New Chinese Gene-Editing Tools Promise Advances in Medicine, Agriculture
(Yicai) Aug. 7 — Chinese researchers have developed two advanced DNA editing tools that could lead to breakthroughs…
Novel Human Genetics Evidence Confirms Estimates of Genetic Prevalence, Underdiagnosis, and Potentially Greater Symptom Burden of Gain-of-Function CASR Variants Associated with ADH1
BridgeBio Pharma, Inc. – By examining over 100 unique variants associated with the CASR gene causative of ADH1,…
8 babies born with DNA from 3 people in world-first IVF trial
Eight healthy babies have been born in the U.K. using a new IVF technique that successfully reduced their…