Position Title
Professor and Chair of Physical Medicine & Rehabilitation
Professor of Pediatrics
Director, MDA Neuromuscular Disease Clinics
- Department of Physical Medicine & Rehabilitation, School of Medicine
- Department of Pediatrics
- MDA Neuromuscular Disease Clinics
Clinical Interests
Dr. McDonald is a pediatric physical medicine & rehabilitation physician who is also board-certified in neuromuscular medicine and pediatric rehabilitation medicine. He is an internationally recognized expert in clinical management, rehabilitation, and precision therapeutics for children and adults with neuromuscular diseases.
Dr. McDonald has been a pioneer in the development of novel outcome measures for clinical trials focused on disabled populations. He is widely known for his expertise in the treatment and evaluation of children and young adults with Duchenne muscular dystrophy and other neuromuscular diseases.
McDonald directs the NIDRR-funded Rehabilitation Research and Training Center in Neuromuscular Diseases and serves as principal investigator of the UC Davis NINDS-funded site in the NeuroNEXT Neurosciences Clinical Trials National Consortium. He is recipient of over $16 million in federal grant support over the past 12 years.
McDonald has been involved in a multitude of NIH, MDA, CDC and Shriners hospitals national committees and serves as a consultant to the pharmaceutical industry regarding clinical trials in Duchenne muscular dystrophy and other muscular dystrophies. He has served on many NIH study sections reviewing research proposals relating to neuromuscular diseases and rehabilitation sciences.
Research/Academic Interests
Dr. McDonald's research has focused on novel clinical endpoint development in neuromuscular diseases, clinical trials and natural history studies in muscular dystrophies, metabolic syndrome, and energy expenditure in childhood disabilities. His work has led to the identification of genetic polymorphisms predictive of clinical disease progression.
Dr. McDonald leads the international Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study. His work has contributed to the development of precision-based therapeutics for DMD and the first two approved therapies targeting the underlying cause of Duchenne muscular dystrophy – dystrophin gene abnormalities leading to absence of the sarcolemmal-associated muscle protein dystrophin. These therapies have been recently approved by the U.S. Food and Drug Administration (FDA) in the case of eteplirsen, and European Medicines Agency (EMA) in the case of ataluren.
Dr. McDonald serves as the national principal investigator for the Capricor multicenter HOPE-2 trial – the first clinical trial of a systemic stem cell therapeutic conducted in DMD. He has also served as an expert for Biomarin, Sarepta, and PTC Therapeutics at the first three FDA Advisory Committee meetings held for Duchenne muscular dystrophy therapeutics.