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I have heard about a therapeutic trial funded by the Medical Research Council and AVI Biopharma on molecular patches. What does this involve?

April 26, 2017AVI biopharma council heard medical research Molecular patches therapeutic trial

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I have heard about a therapeutic trial funded by the Medical Research Council and AVI Biopharma on molecular patches. What does this involve?

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A group of scientists (MDEX Consortium) who work very closely with the three charities involved in DMD in the UK, Action Duchenne, Duchenne Family Support Group (DFSG) and the Muscular Dystrophy Campaign (MDC), secured funds towards a further genetic therapy trial in DMD using molecular patches from the Medical Research Council (MRC) and AVI-Biopharma. The aims of this project were to perform a dose ranging study of intravenously administered patches in a group of 18 DMD children to test the safety and the efficacy of this approach. Efficacy will be measured by checking at the end of the study if functional dystrophin is produced in these boys after treatment.