Scientific Advisory Board

Carsten Bönnemann, MD

SMAB Chairman

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

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.

Anne Bang, 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.

Alan Beggs, PhD

Peter Yurchenco, PhD MD

Peter Yurchenco received his Ph.D. and M.D. degrees from the Albert Einstein College of Medicine in 1975 and 1976. After completing a medical internship at Dartmouth-affiliated hospitals in 1977, he embarked on a residency in Anatomic Pathology with research fellowships in membrane biology and basement membranes at Yale University. In 1984 Dr. Yurchenco joined the Pathology Dept. at the Robert Wood Johnson Medical School in New Jersey (now part of Rutgers University). He is currently a full tenured professor and Director of Experimental Pathology. Two relevant diseases of interest are laminin-deficient muscular dystrophy and Pierson syndrome. Recently the laboratory developed laminin-binding linker proteins that can be used to repair structural defects of basement membranes. One of these proteins was used to repair mouse models of a laminin-deficient congenital muscular dystrophy. The studies, conducted in collaboration with Dr. Markus Reugg (Univ. Basel), have provided groundwork for developing therapies using somatic gene delivery of linker protein DNAs.

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. In 2011, Dr. Chan became the Senior Research Manager at Ultragenyx Pharmaceutical, and in 2018, became Head of Medical Research at TWi Biotechnology

Yiumo Michael Chan, PhD

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.

Francesco Muntoni, MD

Wayne Ogata is the CEO of Asahi Medical Technologies, Inc. He has been a supporter of Cure CMD since 2009 and has been on it's Scientific Advisory Board since 2013. 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 CMD in 2008. Wayne shares in the mission of Cure CMD to improve the lives of those living with CMD and hopes to contribute by focusing on practical treatments and eventually a cure.

Wayne Ogata

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.

Markus Rüegg, PhD

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