The Cooperative International Neuromuscular Research Group (CINRG) is a consortium of medical and scientific investigators from academic and research centers who share the common goal of improving the lives of patients with neuromuscular disease and their families. The CINRG network joins together over 25 clinical and research sites from around the world to perform clinical studies in neuromuscular disorders. The group has successfully enrolled more than 1,200 study participants (predominantly children) into 18 studies to date. Heather Gordish-Dressman, Ph.D., serves as the principal investigator (PI) at Children’s National for both the CINRG Duchenne and Becker Natural History Studies.
The Duchenne Natural History Study (DNHS), chaired by CINRG PI Craig McDonald, M.D., of UC-Davis, has completed funding through a combination of government (National Institute on Disability and Rehabilitation Research at NIH and DOD), foundation (Parent Project Muscular Dystrophy), and industry partner grants. It is the largest natural history study of DMD to date, with a wealth of data that provide natural history controls for both the design of industry trials and the interpretation of clinical trial data for many clinical and biochemical endpoints. The value of these data is recognized in the muscular dystrophy research community globally, resulting in increased interest in confidential access to the data. Dr. Gordish-Dressman continues this effort as lead statistician and has authored several publications in 2017 including a seminal manuscript published by the Lancet in November 2017.
The Becker natural history study, is led by PI Paula Clemens, M.D., in Pittsburgh. The study has reached its enrollment goals and is collecting long-term data on participants with Becker muscular dystrophy. The CoQ-10/Lisinopril clinical trial, funded by the DOD and led by Dr. Clemens is continuing with long-term follow-up of participants. Dr. Gordish-Dressman is currently analyzing data from both of these important studies.