Scientific Advisory Board

ALAN H. BEGGS, PHD

Alan Beggs is director of The Manton Center for Orphan Disease Research at Boston Children's Hospital and the Sir Edwin and Lady Manton Professor of Pediatrics at Harvard Medical School.  He received his AB. degree in biology at Cornell University and his PhD in human genetics at Johns Hopkins University.  Dr. Beggs has been a standing and ad hoc member of numerous National Institutes of Health study sections and grant reviewer for the Muscular Dystrophy Association, March of Dimes and various international funding agencies.  He is a member of the scientific advisory boards of A Foundation Building Strength, the Congenital Muscle Disease International Registry, and several biotechnology companies developing treatments for rare diseases, as well as on the Board of Directors of American MedChem, a nonprofit devoted to drug development for rare and neglected diseases of children.  His laboratory has led studies to identify the genetic basis for a wide variety of rare conditions with a particular focus on skeletal muscle and its defects in congenital myopathies and related neuromuscular diseases.  Current research is focused on utilizing genetic approaches in human patients and animal models to understand the pathophysiology of, and to develop targeted therapies for, these devastating childhood disorders.

RANDALL L. CARPENTER, MD

Dr. Carpenter has over 30 years' experience in academic medicine, basic science and clinical research, and pharmaceutical drug development. He is currently Chief Scientific Officer or the Rett Syndrome Research Trust. His prior experience in the pharmaceutical and biotechnology industries include Co-Founder, President and CEO of Seaside Therapeutics, President and CEO of Sention, VP of Clinical Research & Development and Regulatory Affairs at Adolor Corporation and member of Astra Pain Control's Global Therapeutic Area Team. While in industry, Dr. Carpenter led translational medicine teams responsible for 8 successful IND submissions and dozens of GCP-compliant clinical trials. Prior to joining industry, he held academic faculty appointments at Virginia Mason Medical Center, the University of Washington and Wake Forest University where he specialized in anesthesiology, pain and translational medicine. Dr. Carpenter is the author of more than 70 peer-reviewed articles, holds several patents and has served as editor-in-chief or on the editorial boards of several medical journals. Dr. Carpenter is currently a member of the Scientific Advisory Board of EU-AIMS and the External Advisory Board for the Translational Neuroscience Center at Boston Children's Hospital.

STUART J. SWIEDLER, MD, PHD

Stuart J. Swiedler is a biotechnology consultant with more than 25 years of experience in the biotechnology field, primarily as a scientist and executive at Glycomed, Inc., and as a clinical scientist and senior executive at BioMarin Pharmaceutical Inc. Over a 10-year period at BioMarin, he contributed to both the non-clinical and clinical aspects of drug development for the regulatory approvals of the orphan drugs Aldurazyme, Naglazyme and Kuvan. He continues to focus on orphan drug products for genetic diseases, serving as Chairman of ArmaGen, and providing his clinical expertise to both biotech companies and investment firms. He received his Bachelor of Science degree from the University at Albany, and his MD and PhD degrees from the Johns Hopkins University School of Medicine. He completed a residency program in Anatomic Pathology at Yale University School of Medicine and Duke University School of Medicine, and a post-doctoral fellowship at Duke.