Tag: <span>Duchenne Muscular Dystrophy</span>

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Promising new therapy spares muscle loss in Duchenne muscular dystrophy

A promising therapy for Duchenne muscular dystrophy (DMD) developed by University at Buffalo researchers is moving closer to use in humans. The peptide the new drug is based on was originally found in the venom produced by the type of tarantula that Sachs is holding here.  Credit: University at Buffalo Published in July in Neuromuscular Disorders, the new UB research demonstrates that the novel...

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Cell therapy improves heart function, upper limb strength in Duchenne muscular dystrophy

After boys and young men with Duchenne muscular dystrophy received cardiac progenitor cell infusions, medical tests indicated that the patients’ hearts appeared improved, results from a new study show. Patients in the study also scored higher on arm strength tests after receiving the cell infusions. Results from the HOPE Duchenne randomized clinical trial of 25...

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Brothers with rare terminal muscle disorder fear their future after the FDA denies the drug that keeps them walking

Elliot Johnson, 14, and Henry, 11, both suffer from Duchenne muscular dystrophy It is a rare form of the disease that causes muscle weakness, eventually leading to complete loss of mobility with a mortality rate of around 25 The brothers have been part of a clinical trial with a drug that has shown profound improvements and...

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Gene transfer corrects severe muscle defects in mice with Duchenne muscular dystrophy

Duchenne muscular dystrophy is a rapidly progressive disease that causes whole-body muscle weakness and atrophy due to deficiency in a protein called dystrophin. Researchers at the University of Missouri, National Center for Advancing Translational Sciences, University of Washington, and Solid Biosciences, LLC, have developed a new gene transfer approach that uses an adeno-associated virus vector...

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Heart drug improves or stabilizes heart function in Duchenne muscular dystrophy

Human heart.    Researchers at The Ohio State University Ross Heart Hospital and Nationwide Children’s Hospital have shown early treatment with the heart failure medication eplerenone can improve heart function in young boys with Duchenne muscular dystrophy (DMD) and stabilize heart function in older boys with the disease. The results of their study are published...

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