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.
Tag Archive: Brynn Connor
At 2 ½ years old, pediatricians at Children’s Hospital of Philadelphia diagnosed Brynn Clare Connor with Rett syndrome, a rare neurodevelopmental disorder that disrupts a child’s communication, coordination, muscle control, and growth over time. Caused by a mutation in the MECP2 gene, Rett syndrome can occur in both genders but almost exclusively affects girls.