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Children’s Hospital of Philadelphia Researchers Announce Promising Results from First-of-its-Kind, Multicenter, 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 data on the results of the RP-A501 Phase 1 trial, presented at a late breaking session today at the American Heart Association Scientific Sessions 2024 in Chicago, were also published in the New England Journal of Medicine.

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Dr. Yael Mossé Named a Recipient of the 2024 Penn Medicine Awards of Excellence

Yael Mossé, MD, received the William Osler Patient Oriented Research Award, which was established in 1996 to honor Osler, a “Father of Clinical Medicine” who, in the 1800s, revolutionized clinical teaching and patient-oriented research at the Perelman School of Medicine at the University of Pennsylvania.

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Beyond Blood Fall 2024 Newsletter

Family Newsletter from the Comprehensive Hemostasis and Thrombosis Center at Children’s Hospital of Philadelphia.

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