After false starts and incremental gains, scientists crack the problem that made breathing impossible. Will answers come fast enough to save the sick?

President Donald Trump weighed in on domestic terror incidents involving a synagogue attack in Michigan, an IED placed at the mayor's home in New York City, and a shooting at Old Dominion University ...

Shane DiGiovanna was born with epidermolysis bullosa, or EB, a condition he said is described as "the worst disease you’ve ...

New research suggests the erectile dysfunction drug Viagra could help treat a devastating neurological disorder 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 ...

In this video, we explore engaging activities suitable for kids ages 3 and up. The content includes insights into our new merchandise and music playlist, along with answers to viewer questions. #KidsA ...

The Food and Drug Administration on March 10 changed the approval for a version of the prescription drug leucovorin to include people with a very rare genetic condition. FDA Commissioner Dr. Marty ...

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

The FDA narrowed leucovorin's use to a rare disorder, a major shift after Trump and RFK Jr. promoted it for autism.

The federal Genetic Information Nondiscrimination Act prevents health insurers and employers from discriminating against a person based on their genetic information, Rep. Dave Rogers told the ...

It's long been assumed that koalas in southern Australia are genetically unhealthy. A new study finds they're actually recovering, changing how scientists look at genetic risks.

The drug was finally approved in September 2025 through the FDA’s accelerated approval pathway, based on improvement in knee extensor muscle strength, for adults and children with Barth syndrome who ...