Congenital, Hereditary, and Neonatal Diseases and Abnormalities: News and Research on Glycogen Storage Disease Type IIb

Promising results from phase 1 gene therapy trial for Danon disease

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 single gene mutation. The ...