Sarepta therapeutics has announced a further set of preliminary results of a gene therapy trial that is using a harmless virus to deliver a small but functional dystrophin gene (micro-dystrophin) to people living with Duchenne.

Four people living with Duchenne have now received the potential treatment. High levels of the micro-dystrophin protein were produced in the muscles of all participants and levels of creatine kinase in the blood (a measure of muscle damage) fell while their NorthStar assessment scores increased. Most importantly, no severe side effects have been observed (although some less serious effects have been identified).

Let us once again be reminded that as encouraged as we are, these are preliminary results and we must continue to follow our initial patients and commence a controlled trial. But also know this, we are investing our energy, resources and creativity to moving the development forward as fast as is possible. – Doug Ingram, President and CEO of Sarepta Therapeutics.

So while these results are very encouraging, it’s important that we remember that there are limitations with a trial like this. The trial is very small, just 4 people who have been monitored for a limited length of time, and it doesn’t include a control group. This makes it more difficult to attribute any effects seen during the trial to the potential treatment.

But although there are limitations, the results really are encouraging and Sarepta has already announced plans to for a larger trial that they hope to start as soon as possible that will address many of these limitations.

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