New preclinical findings from extensive cell and animal studies suggest that a drug already used for a rare kidney disease could benefit patients with some mitochondrial disorders—complex conditions with severe energy deficiency for which no proven effective treatments exist. Future clinical research is needed to explore whether the drug, cysteamine bitartrate, will meaningfully benefit patients.
2016 CRC RE@CH Award winners, Sarah Noon, MS, CGC (left) and Eileen Ford (right)
Not all superheroes wear masks and capes. At Children's Hospital of Philadelphia, clinical research coordinators are the often-unsung superheroes doing important work in plain clothes and in plain sight. A good coordinator makes the process of participating in clinical studies easy and enjoyable for families while capably