A Beaverton family that has spent years searching for a replacement gene for their son’s rare genetic disorder says they are ...

Four years later, the Phams are searching for a cure after their son was diagnosed with a rare neurological disorder, hoping to raise funds for clinical trials.

Shane DiGiovanna was born with epidermolysis bullosa, or EB, a condition he described as “often called the worst disease ...

The move comes months after the Trump administration touted leucovorin as a potential therapy for a broader group of patients with autism symptoms.

By Ahmed Aboulenein and Julie Steenhuysen WASHINGTON, March 10 (Reuters) - The U.S. FDA approved leucovorin, a decades-old ...

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 ...

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

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 ...

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.

Taner, M.D., Ph.D., who leads Mayo Clinic's Precision Medicine in Neurodegeneration Lab, studies complex genetic interactions in Alzheimer's disease.

Learn about genetic studies of cognition risks related to Alzheimer's disease in in Mayo Clinic's Precision Medicine in Neurodegeneration Lab led by Nilufer Ertekin-Taner, M.D., Ph.D.

BEAVERTON, Oregon — A Beaverton family that has spent years searching for a replacement gene for their son’s rare genetic disorder says they are seeing encouraging progress. Linda and Tommy Pham say ...