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We are pleased to report that the first ever human exon skipping trial showed positive results. Over the last few months, Prosensa injected three boys with their exon skipping compound into a small section of a single muscle. The objective was to “trick” the muscle cells into producing the dystrophin protein that is missing in Duchenne boys. By using this “genetic velcro” they were able to manipulate the dystrophin gene to produce a shorter, but hopefully functional version of the protein in the small section of muscle in all three boys. Fortunately, no side effects were observed. As this phase of the trial progresses with positive results, Prosensa will apply to the regulatory agencies for a full body, systemic trial. This is a huge move forward but we still have a long way to go before this becomes a reality. Delivering the compound to the all muscles is a difficult hurdle, but we are thrilled to see so many positive steps that give us hope. We are hopeful that we will be reporting more significant and positive news from Prosensa very soon.
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