Alain Geille, 62 old, joined AFM-Téléthon in 2013 as a volunteer and is currently heading the AFM Myotonic Dystrophy nationwide patient group. He retired recently from his engineer and scientist job in Bordeaux, France. He co-organized in 2015 the patient day of the IDMC-10 Paris meeting, and is highly committed in the Myotonic Dystrophy community.
Eugenio Mercuri Md PhD (Associate Professor in Pediatric Neurology): areas of expertise: congenital muscular dystrophy, outcome measures and assessment tools in neuromuscular disorders. Publications: over 130 publications on various topics related to paediatric neurology and neuromuscular disorders.
Kathryn R. Wagner, M.D., Ph.D. is the Director of the Center for Genetic Muscle Disorders at the Kennedy Krieger Institute and Professor of Neurology and Neuroscience at the Johns Hopkins School of Medicine. She received her BS from Yale University and MD and PhD from the Johns Hopkins School of Medicine.
Dr. Wagner’s work focuses on muscle regeneration and therapeutic development for the muscular dystrophies. She is a scientific advisor for the FSH Society, Parent Project Muscular Dystrophy, and SOLID biosciences, chairperson of the TREAT-NMD Advisory Committee for Therapeutics and elected member of American Society for Clinical Investigation.
Dr. Kevin Flanigan is a Professor of Neurology and Pediatrics at the Ohio State University, and a Principal Investigator at the Center for Gene Therapy at Nationwide Children’s Hospital in Columbus, Ohio. He has extensive experience in the design and development of patient registries, and has served as the Director of the United Dystrophinopathy Project, an NIH-funded genotype/phenotype database and registry.
He is a longstanding advocate of transnational efforts in neuromuscular disease, and during a sabbatical at the Institute of Myology in Paris, he helped to develop the minimal data elements of the TREAT-NMD DMD database. He currently serves on the TREAT-NMD Global Database Oversight Committee; the Therapeutic Advisory Committee of TREAT-NMD; the Executive Board of the World Muscle Society; and on advisory committees to establish common data elements for neuromuscular studies for the NINDS. He has extensive experience in clinical trials, and has served on clinical trial advisory committees for PTC Therapeutics, Prosensa, GSK, and AVI. He has appeared before the FDA as part of the IND application process, and has experience in designing IND-enabling pre-clinical programs, including one currently funded by an NIH U01 award directed at gene therapy for MPSIIIB.