Board of Advisors
Our Sincere Thanks to Our Board and Advisors
Our board and advisors allow us to identify the most promising research, conduct due diligence on the researchers, and provide them with ongoing funding and technical assistance. Board members and advisors are pro bono.
Our Board of Directors is composed of five members with many years of collective experience in philanthropy and business management.
- Jeffrey Goffman, Integrated Oncology Network, LLC- Board Chair
- Jesse Dylan, LYBBA, Reform Inc.
- Thomas Liu, Hall & Company
- Debra Miller, President & Founder
- Paul Miller, Founder, Sterling Foods
- Timothy Sandel, Wells Fargo Commercial Banking
- Kathi Sharpe-Ross, Sharpe Public Relations-Marketing LLC
Our Scientific Board of Advisors includes the leading scientists in the world working on muscular dystrophy, and most especially Duchenne Muscular Dystrophy.
Barry J. Byrne, MD, Ph.D.
Professor and Associate Chair of Research, Department of Pediatrics and Microbiology and Molecular Genetics Virginia Root Sutherland Professor of Cardiology Director, Powell Gene Therapy Center Medical Director, Congenital Heart Center University of Florida, College of Medicine
Dr. Byrne is internationally recognized for his work in the areas of cardiomyopathy, transplantation and genetic therapy. His laboratory is actively involved in developing new genetic therapies for cardiovascular disease. In the area of cardiomyopathy, his lab is studying gene replacement in an autosomal recessive form of fatal cardiomyopathy in children. The disease is the prototype of lysosomal storage disorders leading to skeletal and cardiac muscle weakness. The lab has used AAV vectors to achieve sustained correction of the gene deficiency and correction of the phenotype in natural and transgenic mouse models of the disease. The current therapy is currently being proposed for human clinical trials. Similar therapies are being used to combat cardiac transplantation rejection. Secondly, the lab is investigating the ability of mesenchymal stem cells to undergo myocardial specification for the purpose of tissue repair in the heart. Finally, several projects are focused on the use of AAV vectors injected into striated muscle to achieve sustained release of therapeutic proteins, including thrombolytic factors and coagulation factors. These projects are currently funded by the National Institutes of Health (NIH), American Heart Association (AHA), and foundation
Kevin Campbell, PhD
Kevin Campbell is the Roy J. Carver Biomedical Research Chair in Molecular Physiology and Biophysics, Head of the Department of Molecular Physiology and Biophysics, and Director of the Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center at The University of Iowa. Dr. Campbell also holds joint appointments as a professor in the Departments of Neurology and Internal Medicine. In 1989, he was appointed an investigator with the Howard Hughes Medical Institute (HHMI), and in 2009 he received his fourth five-year renewal with HHMI. Dr. Campbell is internationally recognized for his fundamental contributions to muscular dystrophy research. For the past twenty years, Dr. Campbell and his colleagues have actively investigated the molecular pathogenesis of muscular dystrophy. Dr. Campbell’s research is funded by a variety of organizations, and the scientific accomplishments of his laboratory have resulted in plenteous publications in high-quality journals. In addition to receiving numerous awards for his outstanding research, Dr. Campbell is an elected member of the Institute of Medicine, the National Academy of Sciences, and the American Academy of Arts and Sciences. Finally, Dr. Campbell is the co-recipient of the 2009 March of Dimes Prize in Developmental Biology.
Jeffrey Chamberlain, PhD
Jeffrey Chamberlain is the McCaw Chair in Muscular Dystrophy and a professor in the Departments of Neurology, Medicine and Biochemistry at the University of Washington School of Medicine, as well as director of the Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center.
Dr. Chamberlain developed the method of multiplex PCR, commonly used for diagnosis of numerous genetic disorders. He is also a pioneer in the development of gene therapy approaches for the muscular dystrophies. The main focus of Chamberlain’s lab is on the muscular dystrophies, primarily Duchenne muscular dystrophy (DMD), with two major goals: to develop a better understanding of the molecular basis of the pathophysiology of the diseases, and to develop gene and cell therapies with potential to correct and treat the muscular dystrophies. He has published widely on genetics and muscular dystrophy, and is on numerous advisory committees as well as the editorial board of several internationally respected medical journals.
Eric Hoffman, PhD
Chairman, Department of Integrative Systems Biology, George Washington University School of Medicine Clark Chair and Director, Research Center for Genetic Medicine, Children’s National Medical Center in Washington D.C.
Dr. Hoffman has been involved in translational human molecular genetics, and applications of genomics to health problems since the mid 1980’s. Research milestones include identification of dystrophin, defining voltage sensitive ion channel disorders in humans and horses, molecular basis of recurrent pregnancy loss, and treatment of large animal models of Duchenne dystrophy with exon-skipping therapy. His research facility in Washington DC was formed to be translational, and interdisciplinary, with a focus on emerging genome-enabled technologies. Current research efforts include therapeutics of muscular dystrophy, muscle plasticity, diabetes research, and medical rehabilitation. The Center houses 35 faculty, six federally funded Center and Program Project grants, federal training grants in muscle and lung, and works closely with a biotech company (VBpharm) for drug development in neuromuscular disease.
Edward Kaye, M.D.
Group VP Clinical Research at Genzyme
Edward M. Kaye, M.D. is currently Group V.P. and Therapeutic Area Head in Clinical Research at Genzyme Corporation where he supervises the clinical research in the lysosomal storage disease programs and in the genetic neurological disorders. He received his medical school education and pediatric training at Loyola University Stritch School of Medicine and University Hospital, Child Neurology training at the Boston City Hospital, Boston University, and completed his training as a Neurochemical Research Fellow (Geriatric Fellow) at the Bedford VA Hospital, Boston University in 1983. He was head of the section of Neurometabolism, Pediatric Neurology at The Floating Hospital for Children (Tufts University) and research fellow in gene therapy at the Massachusetts General Hospital until 1996 when he moved to Philadelphia to become Chief of Pediatric Neurology and Director of the Barnett Mitochondrial Laboratory at St. Christopher’s Hospital for Children. In 1998, he accepted the appointment as Chief of Biochemical Genetics at the Children’s Hospital of Philadelphia and Associate Professor of Neurology and Pediatrics until moving to Genzyme Corporation at the end of 2001. He continues as a member of the Neurology Department at the Children’s Hospital of Boston and has been on the editorial boards of a number of journals including Annals of Neurology, Journal of Child Neurology, and Pediatric Neurology. He is also on the Scientific and Medical Advisory Boards of the United Leukodystrophy Foundation, Spinal Muscular Atrophy Foundation and Prize4Life
Douglas Macdonald, PhD
Director Drug Discovery at CHDI Foundation, Inc., a biotech company focused on Huntington’s disease, an autosomal dominant genetic neurodegenerative disorder. Macdonald conducts multiple drug discovery programs, including de novo programs at CROs and collaborations with biotechnology and large pharmaceutical companies. Prior to CHDI, he worked at sanofi-aventis, the Schering-Plough Research Institute and at Rockefeller University. He is currently a Lecturer at the University of Southern California School of Pharmacy and Regulatory Sciences and is a member of the NIH/NINDS Neuropharmacology and Diagnostics Small Business Innovation Research Study Section.
Carrie Miceli, PhD
Professor in the Department of Microbiology, Immunology and Molecular Genetics at UCLA.
She is a leading immunologist, focused on T cell receptor signal transduction and T cell biology. She has recently developed an interest in the role of inflammation in DMD pathogenesis. Her research has broad application to cell biology and signal transduction and has helped elucidate molecular mechanisms of T cell activation and tolerance. She has been instrumental in developing a program at UCLA aimed at implementing cellular assays for DMD-directed high throughput small molecule screening. A current focus of this research involves identification of enhancers of anti-sense mediated DMD exon skipping. She is the director of the UCLA Muscular Dystrophy Core Center HTS Core, a member of the Administrative and Executive Committees of the UCLA Muscular Dystrophy Core Center and is a co-director of the Center for Duchenne Muscular Dystrophy at UCLA.
Kannebovina Nagaraju, PhD
Research Center for Genetic Medicine
Stanley Nelson, MD
Professor at the University of California, Los Angeles’ Departments of Psychiatry & Bio-behavioral Sciences, and Human Genetics.
Nelson is trained as a pediatric oncologist, and has developed methods for the genome-wide approaches to study the genetic basis of human diseases. His laboratory has worked in gene discovery in brain cancers, ADHD, autism, and rare mendelian diseases. Recently, he has set up technology to perform complete human genome sequencing. Recently, he has helped to develop the Center for Duchenne Muscular Dystrophy at UCLA, and is co-director of the UCLA Muscular Dystrophy Core Center and director of the bioinformatics/genomics core. His recent work includes the identification of drugs that can enhance the efficiency of antisense based exon skipping therapies.
Pier Lorenzo Puri, MD/PhD
Assistant Professor at the Burnham Institute in San Diego, California.
Puri’s team discovered that ongoing treatment with the deacetylase inhibitor Trichostatin A, currently under clinical study for breast cancer, restored skeletal muscle mass and prevented the impaired function characteristic of muscular dystrophies. Importantly, these restored muscles showed an increased resistance to contraction-coupled degeneration–the primary mechanism by which muscle function declines in Duchenne muscular dystrophy and related dystrophies.
Thomas A. Rando, M.D., PhD
Stanford University School of Medicine
Legal and strategic advisors include:
- Douglas Freeman is a partner in the foundation law firm of Freeman, Freeman & Smiley, and is chairman and National Managing Partner of IFF Advisors. Mr. Freeman assists CureDuchenne in the areas of organizational strategy and development.
- Morrison & Foerster, which has an expertise in biotechnology, is CureDuchenne’s legal counsel. In this capacity, the firm reviews and validates the work of applicants for research funds, and negotiates grant contracts.