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Ulster University Researcher Dr Stephanie Duguez awarded research grant by Muscular Dystrophy UK

Ulster University Researcher Dr Stephanie Duguez awarded research grant by Muscular Dystrophy UK
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New drug molecules hold promise for treating fatal child disease


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Scientists have identified a way to rescue muscle cells that have genetically mutated, paving the way to a possible new treatment for rare childhood illness such as Duchenne Muscular Dystrophy (DMD).
The study, led by the Universities of Exeter and Nottingham, is published in the
Proceedings of the National Academies of Sciences, USA. The research used novel drugs being developed at the University of Exeter, which metabolically reprogram the cellular energy production centres in muscle cells, by providing them with a fuel source to generate metabolic energy.
DMD is a genetic condition caused by a mutation in a gene called dystrophin which results in progressive irreversible muscular degeneration and weakening. Its symptoms include muscle atrophy leading to a loss of the ability to walk in children for which there is no known cure. Currently, the condition is treated with steroids, such as prednisone, but they can stop working and side-effects are com ....

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