Scientific Medical Advisory Board

Carsten Bönnemann, MD

Dr. Carsten Bönnemann is a pediatric neurologist specializing in neuromuscular disorders and neurogenetics. He graduated from medical school in Freiburg, Germany. He completed pediatric training in Hamburg and in Goettingen, and training in child neurology at MGH/Harvard Medical School in Boston. His neuromuscular training continued at Children’s Hospital Boston, where he also conducted research in Dr Kunkel’s lab working on gene identification in the muscular dystrophies. In 2002 he joined the Children’s Hospital of Philadelphia/University of Pennsylvania where he was Co-Director of the Neuromuscular Program and Director of the Neurogenetics Clinic. Since 2010 Dr. Bönnemann is a Senior Investigator in the Neurogenetics Branch and Chief of the Neuromuscular and Neurogenetic Disorders of Childhood Section at the National Institute of Neurological Disorders and Stroke/National Institutes of Health. The clinical focus of the section at NIH ranges from the clinical characterization and management of known and new disorders to translational aspects leading to clinical trials. Research in the lab is concerned with the molecular genetics and cell biology of muscular dystrophies and early onset myopathies in order to develop molecular-based treatments. More basic research focuses on the role of the extracellular matrix in muscle maintenance and regeneration. Before his position at the NIH, Dr. Bönnemann was a Pew Fellow in the Biomedical Sciences and received the Derek Denny-Brown Neurological Scholar Award from the American Neurological Association.

SMAB Chairman

Anne Bang, PhD

Dr. Bang is an experienced cell biologist and stem cell expert who leads efforts to develop patient cell specific and human induced pluripotent stem cell based disease models for drug screening and target identification at Sanford Burnham Prebys Medical Discovery Institute. Her research is primarily focused on neurological and neuromuscular disease, with the aim of designing human cell based models and assays that reflect higher order cellular functions and recapitulate disease phenotypes, yet have the throughput and reproducibility required for drug discovery.

Alan Beggs, PhD

As director of the Beggs Laboratory at Boston Children's Hospital, Dr. Alan Beggs' goal is to study the basic biology of skeletal muscles and to use this information to understand the genes and proteins involved in the cause of neuromuscular disorders. By understanding the cause of neuromuscular disorders, Dr. Beggs hopes to develop better diagnostic tests, treatments and therapies for congenital myopathies.

Yiumo Michael Chan, PhD

Dr. Yiumo Chan’s main research focus is to elucidate the biochemical pathways underlying various forms of muscular dystrophies and to develop treatments for the diseases. He obtained his B.Sc and Ph.D. degrees from the University of Chicago and completed his post-doctoral training in Dr. Louis Kunkel’s laboratory at Children’s Hospital Boston focusing on limb-girdle muscular dystrophies (sarcoglycanopathies). Dr. Chan has been conducting basic and translational research in both sarcoglycanopathies and dystroglyanopathies for the past 10 years. He was a Staff Scientist in Geisinger Medical Center, PA and Senior Scientist in Carolinas Medical Center, NC. Since 2011, Dr. Chan became the Senior Research Manager in Ultragenyx Pharmaceutical, Novato, CA working on other rare forms of myopathies.

John Day, MD, PhD

Dr. Day is a Professor of Neurology and Pediatrics at Stanford University, where he directs the Stanford Neuromuscular Program as well as the pediatric and adult neuromuscular clinics. He has been involved in the diagnosis, management and research of neuromuscular disorders for 30 years, after having trained at the University of California, San Francisco in Neurology, with subspecialty training in neuromuscular disease. Dr. Day founded the Paul and Sheila Wellstone Muscular Dystrophy Center at the University of Minnesota in 1995, and directed it until he moved to California. At Stanford he has created a comprehensive center to optimize care for patients of all ages, with special focus on understanding and treating the multi-systemic features of neonates and infants with muscle disorders, and in developing gene modification methods to conquer these disorders. As a practitioner and investigator he has been involved with Cure CMD since its inception.

Edward Kaye, MD

Ed Kaye is the Chief Executive Officer and a Director of Stoke Therapeutics. Ed served as President and Chief Executive Officer of Sarepta Therapeutics (NASDAQ: SRPT) from September 2016 to June 2017, interim Chief Executive Officer from March 2015 to September 2016, and Chief Medical Officer from June 2011 to April 2017. He also served on the Company’s Board of Directors. Previously, Ed was Group Vice President of Clinical Development at Genzyme Corporation from April 2007 to June 2011, where he supervised the clinical research in the lysosomal storage disease programs and in the genetic neurological disorders. Prior to this, Dr. Kaye held various roles at Genzyme Corporation since 2001, including Vice President of Medical Affairs for Lysosomal Storage Diseases, Vice President of Clinical Research and Interim Head of PGH Global Medical Affairs. Ed currently serves as a member of the Boards of Directors of Cytokinetics, Inc., The Massachusetts Biotechnology Council and Neurovia.

Prior to entering the biotech industry, Ed trained in Pediatrics, Pediatric Neurology, and Biochemical Genetics. He was on the research staff of the Massachusetts General Hospital, Tufts University Medical Center and was the Chief of Biochemical Genetics at the Children’s Hospital of Philadelphia. He continues on staff in Pediatric Neurology at the Boston Children’s Hospital. Ed earned his B.S. in Biology from Loyola University and earned his M.D. at Loyola University Stritch School of Medicine.

Dione Kobayashi, PhD

Dr. Kobayashi is an accomplished executive scientist and President of AkeOla LLC. She has a deep knowledge of and experience in drug development activities for rare diseases, including drug screening, biomarker development and regulatory strategy that spans nearly 20 years. Prior to founding AkeOla, Dr. Kobayashi worked as an executive scientist in several biotech and pharmaceutical companies as well as non-profit organizations including Cydan Development, Alector, the Spinal Muscular Atrophy Foundation, Rinat (a subsidiary of Pfizer), Elan Pharmaceuticals, and Genentech. She has published various journal articles focused in neurodegenerative, neurodevelopment, neuromuscular, and ocular rare genetic diseases.

 

Dr. Kobayashi received her BSci in Brain and Cognitive Science from MIT, MSci in Cell, Molecular and Neurosciences from University of Hawaii Manoa, and her PhD in Neuroscience from the University of Edinburgh. She is a member of the Cure CMD Scientific Advisory Board (SAB), the Bluefield Project to Cure Frontotemporal Dementia Internal Advisory Board, and the Myonexus Therapeutics SAB.

Daniel Michele, PhD

Dr. Daniel Michele is a Professor & PhD Graduate Program Chair in the Departments of Molecular and Integrative Physiology, and Internal Medicine-Cardiovascular Medicine, at the University of Michigan.   His PhD thesis training on genetic cardiomyopathies at the University of Michigan was followed by a post-doctoral fellowship at the University of Iowa, where he worked on the early discoveries of dystroglycanopathies.  As a Biological Sciences Scholar at the University of Michigan, his research has been focused on muscle and cardiovascular disease in dystroglycanopathies and related muscular dystrophies.  He is also Director of the NIAMS supported Physiology Phenotyping Core which specializes in physiological testing of preclinical mouse models of cardiovascular and muscle diseases.  

Professor Francesco Muntoni

Professor Francesco Muntoni is a pediatric neurologist with an interest in clinical and molecular aspects of neuromuscular disorders. As director of the Dubowitz Neuromuscular Centre at Great Ormond Street Hospital, Prof. Muntoni follows one of the largest cohorts of congenital muscle disease patients. His group has contributed to the identification of a number of disease loci and genes involved in 13 neuromuscular disorders in collaboration with European and USA collaborators, including the identification of mutations in the FKRP and LARGE genes. Prof. Muntoni is co-investigator of Duchenne muscular dystrophy exon skipping trial.

Wayne Ogata

Wayne Ogata is co-founder and CEO of RetroVascular, Inc., a medical device start-up focused on the use of plasma-mediated ablation for the treatment of various conditions in the body including chronic total occlusions (CTO). Wayne holds a degree in Bioengineering from UC San Diego. He resides in California with his wife Yukari and two children, Tracy and Alex. Alex was diagnosed with Collagen VI Congenital Muscular Dystrophy (Ullrich) in 2008. Wayne shares in the mission of CureCMD to improve the lives of those living with CMD and hopes to contribute by focusing on practical treatments and a cure.

Markus Rüegg, PhD

Dr. Markus Rüegg is Professor for Neurobiology at the Biozentrum, University of Basel, Switzerland. He studied Biochemistry and Neurobiology in Zurich and Stanford and was co-founder and member of the management of MyoContract, now called Santhera Pharmaceuticals, a biotech company that dedicated to develop therapies for neuromuscular diseases. Dr. Rüegg is an internationally recognized expert in neuromuscular research and has published numerous scientific papers in the field of neuromuscular research. In recent years, his work has also been devoted to the study of therapeutic interventions in mouse models for congenital muscular dystrophy. Dr. Rüegg is also partner of the network TREAT-NMD.

Volker Straub, MD

Dr. Volker Straub, Professor of Neuromuscular Genetics, was trained as a Paediatric Neurologist at the University of Düsseldorf and the University of Essen in Germany. After his PhD thesis on Duchenne muscular dystrophy he worked as a postdoctoral research fellow in the laboratory of Kevin Campbell at the University of Iowa. In 2003 Volker joined the Institute of Human Genetics of Newcastle University, UK. Volker is joint co-ordinator of TREAT-NMD (www.treat-nmd.eu), executive board member of the World Muscle Society and executive board member of the Institute of Human Genetics at Newcastle University. Together with Hanns Lochmüller, Volker was responsible for setting up the German muscular dystrophy network, MD-NET, of which he was joint coordinator until 2008. Within the neuromuscular research group at Newcastle, Volker has a long-standing interest in the pathogenesis of muscular dystrophies, with research using zebrafish and mouse models. His current research also involves the application of contrast enhanced MRI.

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