Deepest thanks to you, the PPMD family, for coming together to speak in One Voice over the past year. I had one conversation this year that really epitomized the idea of our community speaking as one voice . I’ve shared it with you through the Fotobabble video below.
I hope you'll watch my message of hope and consider adding your own
Add to our voice, and create your own Fotobabble “card” to share what you’re thankful for this year. It’s easy.

It is because we have worked together that we’ve accomplished everything we have in 2010. Just look at what our community can do when we speak with One Voice:

• We published the Care Considerations to guide the experts, the primary healthcare providers, and all families.
• We are organizing a dissemination plan for the Care Considerations so that all children, all over the world, will have access to a higher standard of care.
• We gathered in Washington to discuss with Congress our progress to date and to enlist their help to provide FDA resources and expertise and to support a position within FDA for rare disease.
• We responded to Elizabeth McNeil, MD (OOPD)’s idea to organize a workshop with NIH and FDA to discuss exon skipping and how we might work together to expedite exon skipping for rare mutations.
• We were able to provide block grants to clinical sites to facilitate recruitment for Acceleron’s ACE 031 trial and that plans are underway to provide block grants for additional sites in other clinical trials in 2011.
• The Duchenne Therapeutic Development Meeting was added to our already comprehensive Annual Connect Conference, providing a scientific track that hosted some of the most brilliant minds in Duchenne research and care.
• We promoted and participated in the Treat NMD TACT review.
• We organized a Task Force with healthcare providers and other neuromuscular disease groups to promote early diagnosis.
• We facilitated and provided support for the development of an access program for ataluren.
• We partnered to support the Follistatin gene therapy trial at Ohio State University.
• Larry Gold and SomaLogic are providing the opportunity to partner in the development of a biomarkers program for Duchenne.
• We organized and supported workshops concentrating on gaps in Care – endocrinology, cardiology, psychological success factors.
• Our advocacy—your voice in Washington—resulted in two new Wellstone Centers focused on Duchenne: Nationwide Children’s Hospital in Columbus (focusing on gene therapies for Duchenne) and University of Pennsylvania/UCLA (focusing on fibrosis).
• We provided support for two fellowships through the End Duchenne Grant Award Program, which continues to support the most promising work in Duchenne research.

In 2011, we look forward to addressing challenges in the Duchenne community with continued passion and tenacity. Parent Project Muscular Dystrophy, as always, will be investing in research that will continue to bring us closer to treatments and a cure. We will focus on cardiac health and intervention because the heart is a muscle too. We will expand our upon our already robust advocacy agenda. And we will continue to address the gaps in care, so that all our loved ones with Duchenne can thrive.

Thank you for a great year thus far. I hope you and your family have a wonderful Thanksgiving and again, please know how grateful we at PPMD are to have you in our community.

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