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Trails
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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.

Young girl studying drinking an energy drink
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Researchers at Children’s Hospital of Philadelphia Advocate for Energy Drink Regulation

Energy drinks, marketed for boosting energy, concentration, and athletic performance, can pose significant cardiovascular risks, including death. Despite the American Academy of Pediatrics’ advice against caffeine consumption by children and adolescents, the United States remains the least regulated country and should take immediate steps to protect vulnerable populations, leading cardiology researchers at Children’s Hospital of Philadelphia (CHOP) urged in a recent commentary in the Journal of Pediatrics.

Mother hugging distraught daughter
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How to Help Children Grieve

Guiding children through the grieving process allows them to learn healthy ways to deal with their emotions when a loved one dies.

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