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.

Yentli Soto Albrecht, who is a genetic carrier of ALS, is working on research at Penn that could one day prove life-saving.

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

Stanford Medicine pediatric hematologist Agnieszka Czechowicz, MD, Ph.D., has devoted her research career to improving ...

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 ...

Autumn, from York, has a rare genetic condition and underwent six months of treatment in Newcastle.

The FDA has approved a generic drug for a rare genetic disorder. It's called Leucovorin and can be used to treat a brain disorder that limits the delivery of folate, a form of vitamin B, in the body.

In September, Trump and the FDA commissioner announced the drug was under review to benefit patients with autism. The FDA didn't find evidence of that.

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 ...