McArdle disease, also known as glycogen storage disease type V, is an inherited metabolic disorder marked by a deficiency of myophosphorylase, the enzyme essential for the breakdown of glycogen in ...

Researchers at Children's Hospital of Philadelphia (CHOP) announced encouraging results from the first ever gene therapy trial for Danon disease (DD), a rare, X-linked heart condition caused by a ...

In a global first, UConn Health, in collaboration with Connecticut Children's, has dosed a patient in a groundbreaking clinical trial testing gene editing technology to treat glycogen storage disease ...