Parent Project Muscular Dystrophy

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Dear Friend,

We’re so close to reaching our goal of $300,000. Thanks to Ellen Wagner and her family, if you give by the 31st, your donation will go twice as far!

Sharon, our Vice President for Research, wrote to you last week, explaining how important it is to support projects that create meaningful outcome measures for Duchenne therapies. Many people in our community have responded with generous donations.

Please take a minute to share this with anyone in your life who knows what Duchenne is and has asked how they can help us end it. Remember: All online donations up to $50,000 will be matched 100%!

The new year is approaching, and there’s no better way to end 2012 than by reaching our ambitious fundraising goal. Thank you for helping to make it all possible!

Enjoy a bountiful 2013!



A critical moment for our community

Dear Friend,

Take advantage of our matching gift to support vital research that helps all people with Duchenne.  DONATE NOWClinical trial. These two words do something to all of us. We see them and think, "What if this is the one? What if this is it?" Now, with 18 different clinical trials actively recruiting, the chances are better than ever that one of them will be "it." Two words that hold such promise and hope, but also so many unanswered questions.

Our community has hoped for this kind of chance since before PPMD was around. And now we are at the crossroads of possibility. But we need your help—and a generous donor has offered to match your gift through the end of the year. That means your donation will go twice as far to advance crucial projects.

This year, PPMD is taking a unique approach with our holiday campaign. As Vice President for Research, I'm in charge of making every research dollar we spend have the greatest possible impact. We've had a busy year, funding potential therapies that have shown real promise. And in order to make these efforts a success, we need to build the tools that will help us get new therapies approved faster.

This year, to support all of the fantastic trials currently in progress, we're focusing on identifying "biomarkers" and new trial endpoints. We want to ensure that the many clinical trials that are underway or in development will have the greatest possible chance of success. We want to improve the clinical trial process—Better, Faster, Now.

We've had overwhelmingly positive responses from our industry partners to this year's projects. Here's a sample of what we're hearing:

"The projects selected for this year's holiday campaign demonstrate PPMD's deep understanding of drug development and what's required to move the needle. They have supported us since the beginning, and as we head into the next exciting phases of eteplirsen, we are proud to call them partners."

— Chris Garabedian, President & CEO of Sarepta Therapeutics

"PPMD understands that developing viable Duchenne therapies is a collective effort. Their funding of work aimed at identifying and developing new biomarkers could play a fundamental part in advancing promising medicines through clinical trials."

— Jon Tinsley, Senior Director of R&D of Summit plc

"PPMD continues to make informed and highly impactful funding decisions. Their support last year to Tivorsan has helped get us to where we are today. Focusing on projects that will provide companies like ours with important outcome measures is vital to demonstrating the biological activity of promising therapeutic candidates. We will rely on these outcome measures as we advance into a Phase I program. The work supported by PPMD helps lay the foundation for developing effective treatments for not just Duchenne, but rare diseases as a whole."

— Joel Braunstein, Director & CEO of Tivorsan

I'm so proud of all that we've accomplished this past year, and of the partnerships that we've established. We have so much reason for hope in the new year. With such increased interest and support from the pharmaceutical industry, we're poised for even more breakthroughs next year. But we need to rise to this amazing moment and keep the momentum going, so that when the day comes and a therapy for Duchenne is discovered, it is safe, it is approved, and it is accessible.

Please consider making a gift to PPMD this holiday season to help advance this critical and necessary research. All donations up to $50,000 will be matched dollar for dollar. And your gift will ensure that the words "clinical trial" lead to new treatments—and that an end to Duchenne will be possible.

Thank you for supporting PPMD. I hope you and your family have a wonderful holiday season!


Sharon Hesterlee

Sharon Hesterlee
Vice President of Research

P.S. Take a look at the critical projects your gift will help fund. And remember—any donation you make online will be matched and go twice as far in advancing this important work!


Parent Project Muscular Dystrophy's mission is to end Duchenne. We accelerate research, raise our voices in Washington, demand optimal care for all young men, and educate the global community.

© Parent Project Muscular Dystrophy 2012 - All Rights Reserved.
401 Hackensack Avenue, 9th Floor, Hackensack, NJ 07601

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