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 ...

Danon disease is a rare genetic disorder primarily caused by mutations in the LAMP2 gene, which is crucial for the proper functioning of lysosomes—organelles responsible for breaking down waste ...

Results came from a phase 1 trial of RP-A501 for treatment of Danon Disease and sent shares up 75% on the Nasdaq to more than $56, a five-year high. The surging stock price indicates the market's ...