June 25, 2013 Researchers at Boston University College of Health & Rehabilitation Sciences: Sargent College have identified a combinatorial therapeutic approach that has proven effective in treating muscular dystrophy in a mouse model. The findings, published in Human Molecular Genetics, represent a paradigm shift for the treatment of muscular dystrophy as well as a host of other disabling and devastating muscle diseases. The study was led by Mahasweta Girgenrath, PhD, assistant professor and director of the Muscle Disorders and Regenerative Biology Laboratory at BU Sargent College's Department of Health Sciences. Boston University (BU) researchers and postdoctoral fellows Jenny Yamauchi, Ajay Kumar, Lina Duarte, and Thomas Mehuron were collaborators on this study. Muscular Dystrophy type 1A (MDC1A) is the second most common form of congenital muscular dystrophy. Patients with this disease have poor muscle tone at birth, extremely compromised neuromuscular function, and are rarely able to walk independently. Most patients with MDC1A succumb to a premature death due to either respiratory complications or failure to thrive. Although significant strides have been made towards understanding the molecular and biochemical mechanisms underlying MDC1A, there remains no effective therapy in place to combat this lethal disease. The research team, led by Girgenrath, hypothesized that … Continue reading
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