Infrastructure for Clinical Trials in Duchenne Dystrophy

The Cooperative International Neuromuscular Research Group CINRG is a consortium of medical and scientific investigators and centers focused on improving quality of life of neuromuscular disease NMD patients by cooperatively conducting clinical research studies in NMD. The CINRG coordinating center CC is an infrastructure to support the network in conducting study protocols, standardizing clinical trial methodology, data collection and quality assurance, and performing data analyses. In this award period the electronic database capture system was revamped and implemented for two studies in Duchenne Muscular Dystrophy DMD. CINRGs public website has been revamped. One measurement clinical trial was completed, one large observational study is in follow-up and two studies are in active recruitment. New subcommittees were formed three new sites were added to the network. Eleven presentations were given at national and international scientific and parent advocacy groups. Connections with DMD external groups were strengthened and formalized. With a strong ability to assess which outcomes are more sensitive and reliable to measure clinical change, as this CINRG CC supports, therapeutic approaches can be assessed and either adopted or abandoned quickly. This infrastructure support yields better overall results in research for improving care in DMD in the largest network of institutions for this disease worldwide.