It was a big year for children’s health: We celebrated the U.S. Food and Drug Administration’s approval of the world’s first chimeric antigen receptor (CAR)-T cell therapy this September, followed closely by approval of the very first gene therapy to treat inherited blindness this month — both of which have their roots at Children’s Hospital of Philadelphia and the University of Pennsylvania. But besides the big headline-making breakthroughs (brilliant as they are), we wanted to know what other stories captivated our readers in 2017.
Category Archive: Alex's Lemonade Stand Foundation
It was weekend of firsts on many fronts, as physicians, genetic counselors, nurses, researchers, and families gathered July 21–23 at Children’s Hospital of Philadelphia Research Institute for the inaugural Deciphering Beckwith-Wiedemann syndrome (BWS) conference. BWS is a rare overgrowth disorder involving genetic and epigenetic changes that occur approximately every one in 10,500 births.
Summertime lemonade stands and a trip to Disney World have more in common than just the whimsy of childhood: They are just a few of the places where you’ll find members of the Children’s Hospital of Philadelphia community sharing knowledge to improve pediatric research and treatments.
A new month is about to begin, so it seems fitting that this week’s research highlights have lots of “new” initiatives that we’re excited to report.
Beckwith-Wiedemann Syndrome (BWS) is a condition that occurs when parts of the body grow too large, too fast.
The Children’s Hospital of Philadelphia Matthew D. Weitzman, PhD, is studying the relation of a family of enzymes to cancer.
Dr. Kalish recently received an award from the Alex’s Lemonade Stand Foundation to investigate tumor development associated with the rare childhood overgrowth disease Beckwith-Wiedemann Syndrome.