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: Duchenne muscular dystrophy
From the time Antonio Rosato was diagnosed with Duchenne muscular dystrophy (DMD) at age 4½, his family was eager for him to participate in a clinical research trial. They wanted to give him access to the latest advances in pharmacological and disease management approaches for DMD, an opportunity that Antonio’s uncle Artie, who was diagnosed with the same neuromuscular disease four decades ago, did not have.
Editor’s Note: Our clinical research coordinators at Children’s Hospital of Philadelphia are a unique professional hybrid. Part researcher, healthcare professional, data coordinator, social worker, personal coach, and travel concierge, they go above and beyond their job descriptions in order to help families navigate the complexities of participating in clinical research studies. We invited Joshua Zigmont, RN, BSN, who has been a nurse research coordinator for seven years, to describe a typical day on the job. The first thing he told us is that the most consistent thing about the position is its inconsistency: “You have to be flexible because you never know when a curveball is coming,” Zigmont said. So while no day is ever typical, here is a glimpse of a day in the life of a clinical research coordinator.