Scientists at Sanford Burnham Prebys Medical Discovery Institute and an international team of collaborators have defined a new genetic disease marked by premature aging and deficits in brain function.

Last week, the company said that it plans to file a New Drug Application with the U.S. Food and Drug Administration in ...

Researchers at WashU Medicine and collaborating institutions have developed a novel computational tool that can accurately ...

Lissencephaly is a spectrum of rare, genetic disorders in which the brain fails to develop its hallmark folds. The disorders are often associated with seizures and intellectual disability and ...

Gene editing can repair a DNA error in mice that causes Dravet syndrome, a rare, incurable, and potentially deadly form of ...

Genetic testing can help doctors diagnose some neurological disorders, such as Huntington’s disease. It can also provide insights into a person’s risk of future health conditions. Genetic testing ...

Metabolic dysfunction-associated steatotic liver disease (MASLD) affects nearly one in three adults around the globe. It starts when fat builds up inside the liver’s cells. Over time, this buildup can ...

Genome-wide association studies identify genetic overlap among disorders, providing evidence that their distinctions may be misleading One major difference between psychiatric disorders and purely ...

A group of Queen’s researchers is hoping to use gene therapy and new grant money to cure a handful of rare, usually fatal ...