Johns Hopkins scientists develop mRNA "booster" as potential new way to treat rare genetic diseases marked by too low levels ...
Ensuring every newborn has access to life-saving screening and treatment is both a moral and economic imperative.' ...
Acquired DNA mutations found in the SERPINA1 gene can protect liver cells from damage in patients with alpha-1 antitrypsin ...
Danon disease is a rare but serious cardiomyopathy that drastically reduces life expectancy. Male patients infrequently live ...
The Minnesota Department of Health tests for over 60 different disorders during a newborn screening, and now, two more ...
Polycystic kidney disease (PKD), a family of genetic disorders that causes clusters of cysts to form on the kidney, is among ...
Despite insufficient evidence to support direct-to-consumer genetic testing in routine clinical care, cardiovascular clinicians increasingly face questions about its utility and interpretation because ...
If successful, the genetic modification developed by the Transmission Zero team could provide a long-term solution by ...