Barry Byrne, Director of the Powell Gene Therapy Center at the University of Florida, and Associate Chair of Pediatrics
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Dr. Barry J. Byrne is a clinician scientist interested in a variety of rare diseases, with specific attention to developing therapies for inherited muscle disease. As a pediatric cardiologist, his focus is on conditions that lead to skeletal muscle weakness, cardiac dysfunction and respiratory dysfunction. His research team has made significant contributions to the understanding and treatment of Pompe disease, a type of muscular dystrophy resulting from abnormal glycogen accumulation in the muscle. His current research has focused on developing new therapies using the missing cellular protein or the corrective gene to restore muscle function in Pompe and other inherited myopathies. Dr. Barry Byrne is the Associate Chair of Pediatrics and Director of the Powell Gene Therapy Center at the University of Florida. After obtaining a B.S. degree in Chemistry from Denison University, he pursued his medical education, as well as a Ph.D. in Microbiology and Immunology, at the University of Illinois. He completed his pediatric residency, cardiology fellowship training and post-doctoral training in Biological Chemistry at Johns Hopkins University. Joining the University of Florida in 1997, he has served in a variety of clinical, research and educational roles, and is now the Earl and Christy Powell University Chair in Genetics.

Leslie Leinwand, Chief Scientific Officer, BioFrontiers Institute • Professor CU Boulder
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Leslie Leinwand, Ph.D. is a Molecular, Cellular, and Developmental Biology (MCDB) Distinguished Professor and the Chief Scientific Officer of the BioFrontiers Institute at the University of Colorado Boulder. She was recruited to be Chair of MCDB in 1995. She received her Bachelor’s degree from Cornell University, her Ph.D. from Yale University and did post-doctoral training at Rockefeller University. She joined the faculty at Albert Einstein College of Medicine in New York in 1981 and remained there until moving to Colorado in 1995. She co-founded Myogen, Inc. which was sold to Gilead Pharmaceuticals. She was also a co-founder of Hiberna, Inc., and more recently of MyoKardia, Inc., a publicly traded company founded to develop therapeutics for inherited cardiomyopathies. She is a Fellow of the AAAS, former MERIT Awardee of the NIH, Established Investigator of the American Heart Association and was recently elected to the American Academy of Arts and Sciences and the National Academy of Inventors. The interests of Dr. Leinwand’s laboratory are the genetics and molecular physiology of inherited diseases of the heart and how gender and diet modify the heart. The study of these diseases has required multidisciplinary approaches, involving molecular biology, mouse genetics, mouse cardiac physiology, and the analysis of human tissues. Her teaching was recognized by funding from the Howard Hughes Medical Institute’s Professor Program.

Craig McDonald, Professor of Pediatrics and Physical Medicine and Rehabilitation, and Chair of the Department of Physical Medicine & Rehabilitation at UC Davis
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Craig McDonald, M.D. is a an internationally recognized expert in the clinical management, rehabilitation, and precision therapeutics for children and adults with muscle diseases. He has been a pioneer in the development of novel outcome measures for clinical trials in disabled populations. He is widely known for his expertise in the treatment and evaluation of children and young adults with Duchenne muscular dystrophy (DMD). He leads the international Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study, a collaborative effort which has made seminal contributions to the elucidation of the natural history of DMD and validation of the clinical endpoints now used in clinical trials of DMD throughout the world.

Lee Sweeney, Professor at University of Florida, and Director of the Myology Institute
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Much of Dr. Sweeney’s research program is translational in focus and has produced highly cited research on inherited forms of cardiovascular disease and on the skeletal and cardiac aspects of muscular dystrophy. Dr. Sweeney was elected as a Fellow of the American Heart Association in 2001. He has been the director of a Paul Wellstone Muscular Dystrophy Cooperative Center since 2005, which is now competing for renewal as a University of Florida (UF) Wellstone Muscular Dystrophy Cooperative Center. Dr. Sweeney is actively developing therapeutics for rare diseases that include both small molecule and gene therapy approaches. He serves as a consultant to a number of industry therapeutic development efforts for Duchenne muscular dystrophy and spinal muscular atrophy. Dr. Sweeney is well-known in the popular press for his gene-therapy approaches to permanently block the loss of age-related muscle size and strength in mice. The technique suggests that therapies for humans could reverse the feebleness associated with old age or slow the muscle-wasting effects of muscular dystrophies. Based on the enhancement this creates in healthy young animals, Dr. Sweeney has been widely sought as an expert commentator on the potential for gene “doping” in sports, as well as on the bioethical issues surrounding genetic enhancement. In 2004, this work led to Dr. Sweeney being among those chosen by Esquire Magazine as the “Best and Brightest” in America. Dr. Sweeney is also heavily involved in small molecule therapy development for muscle disease. In 2007, he and his collaborators at PTC Therapeutics (a small NJ biotech company) published the development of a compound (PTC 124 or ataluren) that allows read-through of nonsense mutations (premature stop codons) in a variety of genetic disease models. The drug is in clinical trials for Duchenne muscular dystrophy and Cystic Fibrosis. For this work, Dr. Sweeney was awarded a Hamdan Award for Medical Research Excellence from Sheikh Hamdan of Dubai in 2008. On May 23, 2014, ataluren was granted conditional European approval for the treatment of Duchenne muscular dystrophy (DMD), making it the first approved drug for this disease.