Newswise — There are very few physicians who have influenced the study of Duchenne muscular dystrophy (DMD) as much as Dr. Craig M. McDonald, recipient of this year’s American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM) Distinguished Researcher Award.

The AANEM is the premier nonprofit membership association dedicated to the advancement of knowledge, education, and technology in the fields of neuromuscular (NM), musculoskeletal (MSK), and electrodiagnostic (EDX) medicine. Its annual Achievement Award winners highlight prolific members of AANEM and their contributions to advancing these fields of medicine.

“(Dr. McDonald’s) work has profoundly influenced the way we take care of DMD patients,” the AANEM Awards Committee said in its award letter. “Dr. McDonald deserves formal recognition for the multitude of outstanding contributions he has made to AANEM as well as to research regarding the comprehensive diagnosis, treatment, and management of neuromuscular diseases, with particular emphasis on DMD. He is an internationally recognized expert in the clinical management and rehabilitation of neuromuscular diseases including muscular dystrophies and the development of novel outcome measures for clinical trials. He also is widely known for his expertise in the management and assessment of children and young adults with DMD.”

Dr. McDonald has made seminal contributions as leader of the international CINRG (“Synergy”) Duchenne Natural History Study, which is credited with shedding light to the elucidation of the natural history of DMD; validation of the clinical endpoints now used in clinical trials of DMD throughout the world; the development of novel endpoints to assess more severely affected patients with DMD who are non-ambulatory; identification of genetic modifiers which affect DMD disease progression; and application of proteomics to identify novel candidate biomarkers for DMD clinical trials.

“I never dreamed that we would be in a position over the next 30 years to offer dystrophin restoration treatments to boys and young men that would produce transformative outcomes through direct targeting of the DMD disease pathogenesis,” Dr. McDonald said of his life’s work. “It is incredibly rewarding as a physician scientist to generate natural history data and develop novel clinical endpoints used in clinical trials, and I have been excited to be able to offer my patients precision therapeutics that target the underlying causes of neuromuscular diseases and modifying the course of these complex multisystem diseases.”

Dr. McDonald’s enthusiasm for helping in the realm of pediatrics has been one of his greatest motivators.

“The ability to manage pediatric and adult neuromuscular disease patients through the lifespan has been incredibly rewarding to me and has enabled me to develop long-standing close relationships with my patients and their families.”

His work has contributed to the first 2 approved therapies targeting the underlying cause of DMD – 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 ataluran. He served as an expert for Biomarin, Sarepta Therapeutics, and PTC Therapeutics at the first 3 FDA Advisory Committee meetings held for DMD therapeutics.

Dr. McDonald serves as Chair of PMR and is Professor of Pediatrics and PMR at the University of California Davis Health System and School of Medicine. Dr. McDonald is the Director of Rehabilitation Services and former Director of the Spina Bifida and Pediatric Spinal Cord Injury programs at Shriners Hospital for Children Northern California. He also serves as Director and Principal Investigator of the UC Davis NINDS-funded site in the NeuroNEXT Neurosciences Clinical Trials National Consortium (1 of 2 NeuroNEXT sites on the West Coast). As a Pediatric PMR physician board-certified in neuromuscular medicine, he is the only PMR physician in the country to serve as a PI of one of the 25 NIH-funded NeuroNEXT sites. For the past decade he has also been PI of the UC Davis NIDILRR-funded Advanced Rehabilitation Research and Training Center Grant in Neuromuscular Diseases.  Over a decade ago he established the only ACGME-certified Neuromuscular Medicine Fellowship in the U.S. housed in a Department of PM&R.

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About American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM)

Based in Rochester, Minnesota, the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) is the premier nonprofit membership association dedicated to the advancement of neuromuscular (NM), musculoskeletal and electrodiagnostic (EDX) medicine. The organization and its members work to improve the quality of patient care and advance the science of NM diseases and EDX medicine by serving physicians and allied health professionals who care for those with muscle and nerve disorders.

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