The Muscular Dystrophy Association (MDA) has formed a Medical Advisory Team to provide guidance related to research and clinical care in muscular dystrophy, amyotrophic lateral sclerosis (ALS) and other neuromuscular disorders.
The team consists of three physicians, all experts in these conditions and leading clinicians at an MDA Care Center. They will be helping an effort to establish clinical trials that integrate the MDA’s MOVR (neuroMuscular ObserVational Research), a data hub allowing researchers and clinicians to share disease-related information and protocols.
“We believe that engaging this team of world-class experts across neuromuscular diseases will enable MDA to lead the way in speeding the development of groundbreaking therapies and treatment paradigms that will transform the lives of those we serve,” Lynn O’Connor Vos, the president and CEO of MDA, said in a press release.
Barry Byrne, MD, PhD, will be the team’s chief medical advisor. Byrne is a vocal supporter of newborn screening, and was the first physician to give an approved gene therapy to a person with a neuromuscular disorder in the U.S. He is the associate chair of pediatrics, director of the Powell Gene Therapy Center at University of Florida College of Medicine, as well as director of this university’s MDA Care Center.
“We are just beginning to realize the impact of the current revolution in the treatment of neuromuscular disorders. Newborn screening and access to newly-approved therapies are changing patients’ lives when diagnosed with neuromuscular diseases; the MDA Care Center network is ready to provide these innovative treatments,” Byrne said.
As a pediatric cardiologist, Byrne has been focusing on disorders causing skeletal muscle weakness and abnormalities in cardiac and respiratory function. His team has led groundbreaking work in the use of modified adeno-associated viruses (AAV) to develop gene therapies for Duchenne muscular dystrophy, Pompe disease, Friedreich’s ataxia, and other disorders.
“Disease-modifying therapies are no longer a thing of the future. There is an urgent need to bring innovative care, cutting-edge clinical research and new breakthrough treatments to the community,” Byrne added.
Another team member is Matthew Harms, MD, an expert on ALS and similar disorders. Harms is an associate professor at the Vagelos College of Physicians and Surgeons of Columbia University, and also works at the university’s MDA’s ALS Care Center. He received the MDA’s Diamond Award for his leading role in studies into precision medicine for ALS. His work has also led to advancements in the understanding of limb-girdle muscular dystrophy type 1D.
Notably, Stanford’s Neuromuscular Program has helped pioneer genetic treatments for muscular dystrophy, ALS, and spinal muscular atrophy, as well as data hubs such as MOVR. Day also directs Stanford’s MDA Care Centers, and has been studying the biological processes underlying myotonic dystrophy and other muscular dystrophies.
“These physicians are pillars of the neuromuscular disease community, each with focused expertise in specific diseases,” O’Connor Vos said. “Together, they will provide critical advice to MDA on our innovations in science and care programs and help ensure that we are making the maximum impact as the landscape of neuromuscular research, care and treatments evolve.”
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