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Congenital Hyperinsulinism Center News

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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

Nov 18, 2024

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.

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

Nov 12, 2024

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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