The CINRG network is governed by a by-law document to ensure its' research and clinical activities follow principles of open and full scientific communication, peer review, full disclosure of potential conflicts of interest, and democratic principles. An elected Executive Committee provides guidance and direction for the network and is advised by a Scientific Advisory Committee (SAC). The SAC is composed of independent physicians and scientists from academia, non-profit organizations/foundations and the government with expertise in Neuromuscular Disorders and is appointed by CINRG's Directors. The SAC recommends research priorities and reviews the network's progress on a rolling basis. A Data Safety Monitoring Board, convened at least annually by the Coordinating Center, reviews all active protocols and ensures that all studies are conducted safely and adhere to federal guidelines for safety standards. The DSMB has extensive experience in clinical research and safety monitoring in a variety of disease states, including neuromuscular disorders and includes patient representatives.
CINRG is led by an elected Executive Committee composed of some of the leading researchers in Neuromuscular Disorders plus some of the Principal Investigators from the CINRG Network. This combination of NMD scientific knowledge combined with an in depth understanding of CINRG provides excellent leadership for CINRG. The Executive Committee Members are listed below.
Medical Director: Paula Clemens, MD
Dr. Clemens' activities include patient care, with a specific emphasis on inherited myopathies, clinical research in Duchenne Muscular Dystrophy and late-onset Pompe disease and basic laboratory research. She directs the neurology service of the Pittsburgh PA Healthcare System. She is actively involved in both graduate and medical education.
Dr. Clemens directs an active basic research laboratory focused on gene transfer for the treatment of skeletal muscle diseases. The principal disease models studied in her laboratory are Duchenne muscular dystrophy and muscle cachexia. Her laboratory pursues studies of in utero gene transfer to muscle using both intramuscular and systemic delivery approaches. Her laboratory has a long-standing interest in the immunology of gene transfer for muscle disease. The current direction of this research is to characterize the role of regulatory T cells in the muscle pathology of the mouse model of Duchenne Muscular Dystrophy, with particular emphasis on the setting of therapeutic dystrophin gene transfer.
Dr. Clemens is the study chair for two CINRG studies: PITT0908 Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophy and PITT0112 Becker Muscular Dystrophy A Natural History Study to Predict Efficacy of Exon Skipping. Dr. Clemens has been a member of the CINRG Executive Committee since its inception in 2008. She is also the Chair of the CINRG Publication Subcommittee.
Interim Scientific Director: Kanneboyina Nagaraju, DVM, PhD
Dr. Kanneboyina Nagaraju, DVM, PhD is a tenured Professor of Integrative Systems Biology and Pediatrics at George Washington University School of Medicine and Health Sciences and Interim Director of Research Center for Genetic Medicine at Children's National Health System (CNHS) and acting Chair of the Department of Integrative Systems Biology at George Washington University. In the private sector, he is co-founder of ReveraGen Biopharma, and co-founder and President of AGADA Biosciences. Both companies are engaged in the discovery and development of therapeutics for the treatment of neuromuscular diseases. He is a recognized expert on translational research on muscle diseases. Dr. Nagaraju's research led to the identification of powerful anti-inflammatory compounds that resemble glucocorticoids without side effects. He has received extensive grant support for his research from the NIH, US Department of Defense, and several philanthropic foundations. He currently serves on medical advisory board of the Myositis Association and a core member of TREAT-NMD Advisory Committee for Therapeutics, Medical Advisory Committee of Muscular Dystrophy Association and Scientific Advisory Committee of Cure Duchenne Inc. Dr. Nagaraju has authored several textbook chapters on muscle diseases and has over 100-refereed publications. He has led international efforts to define reliable and sensitive outcome measures for drug efficacy in mouse models of neuromuscular diseases and currently directs a robust basic and translational research program dedicated for neuromuscular disorders at CNMC.
Coordinating Center Director: Avital Cnaan, PhD
Avital Cnaan, Ph.D. has more than 25 years' experience as a statistician in multi-center clinical research studies. For the past 20 years, Dr. Cnaan has been directing coordinating centers of federally funded multi-center clinical research studies. Dr. Cnaan is a Professor of Pediatrics, Epidemiology and Biostatistics at The George Washington University Schools of Medicine and Health Sciences and Public Health and Health Services and Chief, Division of Biostatistics and Study Methodology at Children's National Medical Center. She has been directing the CINRG Coordinating Center for the past four years. Her diverse experience, including serving for fifteen years on the Institutional Review Board (IRB), serving on numerous scientific review committees, data and safety monitoring committees, and on the FDA's Pediatric Advisory Committee provides her with a broad appreciation for the complexities and ethical and regulatory issues in pediatric clinical research and strong experience in the process of protocol development, review, and implementation. Dr. Cnaan has co-authored more than 150 scientific publications in a variety of areas of medical research. Dr. Cnaan is also a Fellow of the American Statistical Association. in the Schools of Medicine and Health Sciences and Public Health and Health Services at George Washington University. Dr. Cnaan is a Fellow of the American Statistical Association.
Royal Children's Hospital, Melbourne, Australia: Monique Ryan, MD
Dr. Ryan is Head of the Neuromuscular Clinic and Neuromuscular Clinical Research Program at the Royal Children's Hospital in Melbourne Australia. She trained as a pediatric neurologist and completed a Neurophysiology fellowship at the Children's Hospital Boston and the Lahey Clinic, prior to returning to Australia in 2003. Since 2003 she has been a CINRG PI, initially at the Children's Hospital at Westmead in Sydney, and now in Melbourne. She has been site PI on a number of CINRG trials in addition to the Ataluren and Prosensa trials in DMD, and is also actively involved in natural history and therapeutics trials in the inherited neuropathies.
Alberta Children's Hospital, Calgary, Alberta, Canada: Jean Mah, MD
Dr. Mah is a pediatric neurologist and director of the Pediatric Neuromuscular Program at the Alberta Children's Hospital in Calgary, Alberta, Canada. She is an Associate Professor in the Department of Pediatrics and Clinical Neurosciences at the University of Calgary. She completed her Pediatrics training at the University of Alberta, her Neurology training at the University of North Carolina, her Neuromuscular fellowship at the Medical College of Virginia, and her Master of Science degree in Health Research from the University of Calgary. She has been a member of the Cooperative International Neuromuscular Research Group since 2005. Dr. Mah is currently involved in a number of collaborative research studies related to pediatric neuromuscular diseases, including the infantile facioscapulohumeral muscular dysrophy study.
Institute of Neurology of Schneider Children's Hospital of Israel, Tel Aviv, Israel: Yoram Nevo, MD
Prof. Nevo is the head of the Institute of Neurology of Schneider Children's Hospital of Israel. Until recently he was the head of Pediatric Neurology Unit at the Hadassah Hebrew University Hospital. His training included an MD degree at the Sackler School of Medicine, Tel-Aviv, Israel. He graduated Pediatrics Residency and Pediatric Neurology Fellowship at the Tel-Aviv Medical Center and Neuromuscular fellowship at Washington University, St. Louis, Missouri, USA. Prof. Nevo is a PI of the Israeli CINRG site since the year 2000. He was previously the chair of therapeutic sub-committee of CINRG. Prof. Nevo's site participated in numerous clinical trials both academic and pharma funded for patients with DMD. He is the head of a Pediatric Neuromuscular Laboratory which focuses on the study of different pathways of muscular dystrophy and the effect of anti-inflammatory and anti-fibrotic medications of in-vitro and in-vivo models of muscular dystrophy as well as modes of delivery to the dystrophic muscle. His research was supported by the AFM, Israeli ministry of health and Israel science foundation, Israeli ministry of science and technology and Teva Pharma company.
Duke University, Durham, NC: Laura E Case, PT, DPT, MS, PCS, C/NDT
Laura Case is an Assistant Professor at Duke University, in the Doctor of Physical Therapy Division in the School of Medicine. She has been a physical therapist for over 30 years, has been a Board Certified Clinical Specialist in Pediatric Physical Therapy since 1993. Laura has dedicated her career to the treatment across the lifespan of individuals with neurological and neuromuscular disorders with their onset in childhood, including Duchenne / Becker and other muscular dystrophies, spinal muscular atrophy, cerebral palsy, Pompe disease; to research in neuromusculoskeletal disorders with their onset in childhood; and to teaching. She has participated in the development of international guidelines for the management of Duchenne muscular dystrophy, Pompe disease, and other glycogen storage diseases. She teaches and consults internationally, has worked on a number of Center for Disease Control (CDC) Task Forces, is active in the Pediatric Section of the APTA, and is a member of the North American Pompe Registry Board of Advisors. She has published a number of peer-reviewed articles and received the Duke Presidential Award for Meritorious Service in 2002. She has been a CINRG Clinical Evaluator since January 2014.
Children's National Health System, Washington, DC: Chris Spurney, MD
Dr. Spurney is an Associate Professor of Pediatrics in the Division of Cardiology at Children's National Medical Center (CNMC) in Washington, DC and also a faculty member in the Center for Genetic Medicine Research in the Children's Research Institute at CNMC. He completed his pediatric residency training at New York-Presbyterian Hospital and his pediatric cardiology fellowship at CNMC. He is active in both clinical and preclinical research and is independently funded by the Muscular Dystrophy Association. He has published both preclinical and clinical based articles on cardiac disease in muscular dystrophies. Dr. Spurney is co-Director of the Mouse Preclinical Testing Facility at CNMC and has overseen preclinical drug trials for the past 4 years. He is also the faculty attending for the Muscular Dystrophy Cardiology Clinic at CNMC for the past three years. Dr. Spurney has worked with the Cooperative International Neuromuscular Research Group (CINRG) network for the past three years. He is a site PI and central reader for a CINRG trial studying cardiac outcome measures and is site-PI for a Lisinopril/Coenzyme Q10 cardiac treatment protocol.
Newcastle University, Newcastle, England, UK: Michela Guglieri, MD
Dr Michela Guglieri is an adult neurologist and consultant at the John Walton Muscular Dystrophy Research Centre in Newcastle, UK. She completed her training at the University of Milan and then moved to Newcastle upon Tyne where she developed a particular interest for clinical and translational research in neuromuscular diseases. In Newcastle, she has been involved in several clinical trials and natural history studies in Duchenne muscular dystrophy, Becker muscular dystrophy, FSHD, SMA, Pompe disease and other genetic muscle conditions. She is the Chief Medical Coordinator of the FOR DMD study, an international multicentre study funded by NIH to investigate the optimum corticosteroid regime for DMD.
Coordinating Center: Andrea Smith, MS, LCGC, CCRP
Andrea Smith is a clinical research coordinator at the University of Pittsburgh with over 5 years of experience working in neuromuscular clinical research. She currently coordinates all aspects of 5 ongoing CINRG studies at the Pittsburgh site including regulatory compliance and participant recruitment and retention. Andrea also functions as a Project Manager for CINRG and brings her experience in research coordination to this position. In addition to her clinical research duties, Andrea also serves as the genetic counselor for the pediatric and adult Muscular Dystrophy Association (MDA) clinics located in Pittsburgh, PA.
Royal Children's Hospital, Melbourne, Australia: Kate Carroll, PhD
Kate is a senior physiotherapist who works in a combined clinical and research role in the Neuromuscular Team at The Royal Children's Hospital in Melbourne, Australia. She has worked in paediatric neurology for twenty years and in 2008 she completed her PhD. Kate has been a CINRG clinical evaluator since 2003 and has also acted as coordinator and PI on CINRG and other research studies. She has been an elected member of the CINRG Outcomes Subcommittee since its inception.