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Orphazyme announces publication of results from its Phase
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Is donation really the best way to prevent mitochondrial disease? » MercatorNet
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Study paves the way to a potential new treatment for rare terminal childhood illness
Scientists at the University of Exeter have identified a way to rescue cells that have genetically mutated, paving the way to a possible new treatment for rare terminal childhood illness such as mitochondrial disease.
The research, funded by the United Mitochondrial Disease Foundation in the USA, was led by Professors Matt Whiteman and Tim Etheridge. In the study, published in the
Journal of Inherited Metabolic Disease, the team used novel drugs being developed at the University of Exeter, which metabolically reprogramme mitochondria - the cellular energy production centres in cells, by providing them with an alternative fuel source to generate metabolic energy in the form of minute quantities of hydrogen sulfide.