"Monogenic" diseases, triggered by mutations in just one gene, may actually be more complex than scientists thought.
Massive Swedish study of over two million people reveals that genetic risk for mental illness often points toward ...
A mother in Laurel, MT is opening up about her battle with a genetic kidney disease and her quest for a donor. Tiffini Gallant has been living ...
After treating the local community for more than 30 years, Dr. Stephen Kemker became a patient himself ...
A new study led by researchers at Vanderbilt Health has identified medical conditions that often precede an Alzheimer's ...
Juxtapid has been available to treat adults with homozygous familial hypercholesterolemia (HoFH), a rare genetic disorder that causes extremely high levels of LDL cholesterol.
Forget Vertex Pharmaceuticals -- CRISPR Therapeutics is a better buy right now.
Baron Capital, an investment management company, released its Q4 2025 letter for its “Baron Global Opportunity Fund”. A copy of the letter can be downloaded here. Baron Global Opportunity Fund ...
Over 27,000 kidney transplants were performed in 2025. Less than 25 percent involved a living donor. A New Orleans woman with a rare disease is praying the odds will lean in her favor.
Researchers surmised that DeepRare had the potential to accurately diagnose a disease much earlier.
Results show that targeting the underlying cause of Dravet syndrome with zorevunersen may improve outcomes for people with this rare, devastating genetic neurodevelopmental disease- ...
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