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Study of Rare Disease Reveals Gene Crucial to Immune Defense

Published on March 13, 2015 in Cornerstone Blog · Last updated 1 month 2 weeks ago
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We’re so pleased to announce that Children’s Hospital’s Robert M. Campbell, MD, was honored with Pennsylvania Bio’s Patient Impact Award at the organization’s annual dinner last night! Dr. Campbell, a professor of Orthopaedic Surgery at the University of Pennsylvania’s Perelman School of Medicine, is the inventor of the vertical expandable prosthetic titanium rib (VEPTR), the first device approved by the FDA to treat thoracic insufficiency syndrome (TIS), a rare condition affecting children in which the thorax cannot support regular growth or breathing.

Pennsylvania Bio’s Patient Impact Award “recognizes a company or organization that has made a significant contribution to the quality of healthcare or length of life of patients in 2014.” Last year, the University of Pennsylvania and CHOP were jointly given the award for their groundbreaking immune therapy research.

Dr. Campbell led a recent Journal of Pediatric Orthopaedics study that showed VEPTR treatment improved Jeune syndrome (a severe form of TIS) patients’ survival to nearly 70 percent, compared to a 70 to 80 percent mortality rate without treatment. To learn more about this inspirational work, check out the video below, the first on CHOP Research’s new YouTube channel!