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 ...

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 ...

Rocket’s RP-A501 for Danon disease has potential to be a $1B commercial opportunity, the analyst tells investors in a research note. The firm thinks an accelerated approval pathway may be ...

Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) stock has hit a 52-week low, dropping to $10.11, as the company faces a ...

Particularly, the lead program RP-A501 for Danon disease shows a strong potential for market success, given its high probability of success and a substantial market opportunity. The agreement with ...

Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) operates as a late-stage biotechnology company that focuses on developing gene therapies for rare and devastating diseases. Rocket Pharmaceuticals, Inc ...