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Encouraging results of UK Duchenne exon skipping
Author:System.Web.HttpCookie Date:2012-07-25

The full results of the UK exon skipping trial for Duchenne muscular dystrophy have been published showing that dystrophin - the protein missing in boys with Duchenne - was produced with no significant side effects. The trial involved delivering a 'molecular patch' (AVI-4658) to the whole body by injection into the bloodstream. Three of the trial participants had a strong response to the 12-week treatment - one had almost a fifth of the amount of dystrophin normally found in healthy muscle. There were also indications that the new dystrophin was working correctly in muscle. However, the response to the treatment was very variable from patient to patient.

The drug tested in this clinical trial was developed by scientists in the MDEX consortium and they conducted this trial with funding from the UK Medical Research Council (MRC) and the pharmaceutical industry. The Muscular Dystrophy Campaign has invested more than one million pounds into the development of this therapy over the past 20 years and was instrumental in setting up the MDEX consortium. We continue to represent the patient's voice as one of the three charities on the MDEX scientific advisory board.