Thanks to everyone who contributed so generously to PPMD’s holiday appeal we have been able to fund three new projects designed to speed the progress of the clinical trials underway in Duchenne. As many of you know, we have a three-pronged research strategy that consists of:

  • "Better"  projects designed to put the best candidates into clinical testing while not wasting time and money on the ones we don’t think are likely to work;

  • “Faster”  projects designed to make clinical trials faster and cheaper; and

  • “Now”  projects designed to take advantage of drugs that are already approved that may have some benefit in Duchenne.



What your holiday donation has helped us fund

With a very full pipeline of new drugs in clinical testing, this holiday season was all about “Faster”! Your dollars helped us fund:

  • Dr. Craig McDonald to collect data that will help us design new ways to measure whether or not a therapy is working, possibly including blood samples;

  • Dr. Krista Vandenborne to determine the ability of magnetic resonance imaging (MRI) to detect changes in the muscles of those participating in the Sarepta trial; and

  • Dr. Berch Griggs and Dr. Katie Bushby to collect additional blood samples during the FOR DMD steroid trial to look for predictors of disease progression and to measure bone health.

Making clinical trials faster and cheaper

These types of projects are important because they can help us decrease the total number of participants that are needed for a given clinical trial and may help decrease the time it takes to measure changes in response to a therapy. The less it costs to develop therapies for Duchenne the more companies become interested in this area and the more shots we get at the finish line.

So, although PPMD does fund a significant amount of new therapy development directly, sometimes it’s equally important to fund the tools and techniques that will give these therapies the greatest chance for success in clinical testing. We believe that is a matter of “when” not “if” for meaningful treatments for Duchenne. Thank you for helping us make that “when” as soon as possible!

Sharon Hesterlee, Ph.D.
Vice President, Research
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