An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

Korean researchers have proposed a new treatment strategy that can increase the effectiveness of the genetic rare disease ...

Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...

Muscular Dystrophy Association Clinical & Scientific Conference, convening in Dallas, Texas, from March 16-18, will feature ...

Most individuals diagnosed with a rare disease won’t live to become teenagers or adults. I knew what I had to do.

The early diagnosis of Alzheimer’s disease (AD) through the detection of p-tau proteins in the blood is less disease-specific than assumed since these biomarkers are also elevated in the blood of ...

The challenge of the obesity epidemic is widespread and one that even pediatricians face constantly,” a Connecticut doctor ...

Prince Frederik of Luxembourg died from a genetic disease called PolG, but experts hope his passing could bring more awareness to the rare condition that impacted his life.

While plenty of people are going green in honor of Saint Patrick's Day, one student at East Stroudsburg University is turning ...