Children’s Hospital of Philadelphia Researchers Announce Promising Results from First-of-its-Kind, Multicenter, Phase 1 Gene Therapy Trial for Danon Disease

Results show potential for gene therapy to improve long-term cardiac outcomes PHILADELPHIA, Nov. 18, 2024 /PRNewswire/ — 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…