The U.S. Food and Drug Administration (FDA) has announced a groundbreaking approval of Kebilidi (eladocagene exuparvovec-tneq ...

The family of little Sebastian Botirius says it was a bittersweet moment on Tuesday night as they surveyed construction of a ...

Haemophilia is a rare bleeding disorder that mostly occurs in men An expert explains why and shares ways to reduce the risk ...

In an update posted today, Neurogene said that the "emerging treatment-related serious adverse event (SAE)" was a case of systemic hyperinflammatory syndrome, a rare and life-threatening immune ...

A 29-year-old local patient is about to undergo a life-changing treatment that could dramatically alter the course of his ...

A new treatment that could prevent blindness in children with the CLN2 type Batten disease has been trialed by clinicians and ...

Researchers at Washington University have discovered a new genetic disorder affecting protein folding, offering potential new treatment avenues for rare brain conditions. When most people feeling ...

I suggest you move to China immediately and see how that works out for you,” the Robeson County senator is said to have ...

The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...

BRUNSWICK COUNTY, NC (WWAY) — Many people are unfamiliar with a disorder called 22q11.2 Deletion Syndrome or 22q. Which is ...

Male specimen of angular rough shark with leucism (Andrej A Gajić et al, Journal of Fish Biology) The scientists consider the shark a case of leucism, a rare genetic disorder that affects melanin ...

Saturday Nov. 16 is Cadasil awareness day, a genetic disorder categorized by stroke, cognitive decline, and dementia.