A special space is open on the walls of Adeline Vanderver, MD's, new office for a framed collage of "Commander" Massimo "Mo" Damiani, a toddler wearing an orange jumpsuit who looks ready to join a NASA astronaut team. Dr. Vanderver, a child neurologist and geneticist, helped to pinpoint his rare subtype of leukodystrophy, a group of inherited degenerative diseases that
If scientific research were like building a house, pilot studies would be the foundation. Their purpose is to establish solid evidence that will attract external support for large-scale studies. The Center for Pediatric Clinical Effectiveness (CPCE) at The Children’s Hospital of Philadelphia is giving three investigators the tools that they need to get to start digging.