Researchers at VIB and Antwerp University have identified a major genetic risk factor for a rare form of frontotemporal dementia. The discovery, published today in Nature Genetics, provides a biological entry point for a disease subtype that has been difficult to study.

The FDA proposes new rules allowing mutation-specific gene therapies to qualify for approval despite extremely small patient populations.

Many psychiatric disorders are more biologically related than previously understood, having more genetic similarities than differences, a new study shows. More research is needed to understand how these genetic similarities manifest in different groups of ...

Nick Jonas' neighbour Maya Kibbel recently died of Wilson's disease at the age of 30. The disease is a rare genetic disorder where copper gets accumulated in the body, affecting different organs.

"It's a slow-moving train wreck," Mike Graglia says about his 12-year-old son Tony's rare genetic disease with no cure. Caused by a tiny fluke of nature—a mutation in a gene known as a SYNGAP1—the non-hereditary condition can lead to intellectual ...

New research uncovers how cannabis addiction, not casual use, is genetically tied to severe mental health conditions, raising red flags for policy, treatment, and prevention. Study: The genetic relationship between cannabis use disorder, cannabis use and ...

Hunter Syndrome is a rare genetic condition that can cause damage to the body and the brain. It often causes children to pass away in their teens, but a UNC Health

Sildenafil—an active ingredient also marketed under the name of Viagra—improves symptoms in patients with Leigh syndrome. This has now been reported in the journal Cell by researchers at Charité—Universitätsmedizin Berlin,

If you haven’t heard of the genetic disorder Heterotaxy, a new children’s book released by a Connecticut teacher and her cousin is spreading awareness. Connecticut