Tobi Jesse turned 4 years old on Christmas Eve. He lives with autism and a rare, progressive neuromuscular disease called ...

An experimental gene therapy seems to slow the progression of Huntington’s disease by about 75 per cent, and researchers are ...

An American Heart Association presidential advisory in 2023 introduced a new term: cardiovascular-kidney-metabolic syndrome.

A deadly deer disease is quickly spreading across areas of Middle Tennessee, inching its way closer to Nashville and Davidson County.

Search is ongoing for stem cell donor for little boy, but 12 people may have found matches to their serious diseases.

NEW YORK – BioMarin has stopped developing the investigational BMN 349 for treating alpha-1 antitrypsin deficiency (AATD)-associated liver disease, the company announced in a regulatory filing this ...