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.

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.

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

Children’s Hospital of Philadelphia announced today that it has earned the 2025 Military Friendly ® Employer designation. This is the fifth year in a row that CHOP has received the distinction.

Study found statistically significant benefit when health workers made efforts to coordinate asthma care between homes, schools and primary care providers

Made possible with a $4 million grant from the Brazilian Ministry of Health, CHOP cell therapy specialists will offer their expertise and resources to oversee this initiative.

Dr. Lori Handy recommends that you speak with your healthcare provider or travel clinic regarding vaccines or medications important for your international travel destination.

Download a copy of our updated vaccine safety booklet. Check out recent data about vaccine rates among kindergartens and what it means.

Find info about primary vs. secondary sources when evaluating information about a scientific study.

CHOP’s Center for Fetal Diagnosis and Treatment and Fetal Heart Program, performs fourth successful fetal heart surgery to remove life-threatening tumor.