One-year data from the first four patients dosed is published in JAMA Neurology NATIONWIDE CHILDREN’S HOSPITAL Researchers from Nationwide Children’s Hospital have published in JAMA Neurology results from the first four patients treated in the first clinical trial of systemic delivery of micro-dystrophin gene therapy in children with Duchenne muscular dystrophy (DMD) – and initial...
Tag: <span>DMD</span>
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Cheaper drug just as effective protecting heart in duchenne muscular dystrophy
OHIO STATE UNIVERSITY WEXNER MEDICAL CENTER COLUMBUS, Ohio – A new clinical trial conducted at The Ohio State University Wexner Medical Center found a cost-effective generic medication works just as well as a more expensive drug in preserving cardiovascular function in boys with Duchenne muscular dystrophy (DMD). Results of this multi-center trial are in today’s...
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Gene editing enables researchers to correct mutation in muscle stem cells in DMD model
by University of Missouri Duchenne muscular dystrophy (DMD) is a rare but devastating genetic disorder that causes muscle loss and physical impairments. Researchers at the University of Missouri School of Medicine have shown in a mouse study that the powerful gene editing technique known as CRISPR may provide the means for lifelong correction of the...