view this email online
Parent Project Muscular Dystrophy Donate Now
PPMD to Fund $1 Million in Exon Skipping
August 2013
Facebook Twitter RSS Feed Youtube

Some of us are just getting over saying goodbye to another summer. Others are winding down, holding on to the last couple of weeks. I, myself, just spent a week with family at a lake retreat in Michigan I went to as a child. It was great to spend time with my girls, my husband, and nature. It was also great to recharge.

As we head into Fall, there is an enormous amount of work to do. As progress continues to be made in research, clinical trials, discussions with the FDA, etc., etc., now is not the time to rest. That's why I am proud to share that PPMD has put out a Request For Applications (RFA) and will fund up to $1 million in projects by mid-2014 focused on the development of antisense oligonucleotides or other techniques to skip exons that are not currently in clinical development, or for multi-exon skipping projects, including the skipping of duplications. This is in addition to our ongoing cardiac initiative and other projects we will be announcing soon.

You will also see in this newsletter updates on transition care. As people with Duchenne continue to go off to college, enter the workforce, and marry, it is more important than ever for us to start addressing the needs of adults with Duchenne, while they are still kids.

Speaking of adults with Duchenne, Mitchell Peterson, our Featured Voice for August, is an exceptional young man whose family has supported PPMD since he was diagnosed. This year marks the 15th Annual Mitchell's Run Thru Rockford – the longest running event for PPMD. Meet this amazing 17 year old.

As Ivy writes in her latest blog, it's time to pull the weeds, clear the path, and focus on the task at hand. It's time to end Duchenne.

Enjoy the rest of your summer!

Pat Furlong
PPMD Founder & President

Parent Project Muscular Dystrophy Fund $1 Million in Exon Skipping Projects
PPMD announced that it will fund up to $1 million in projects by mid-2014 focused on the development of antisense oligonucleotides or other techniques to skip exons that are not currently in clinical development, or for multi-exon skipping projects, including the skipping of duplications. A Request for Applications (RFA) has just been released.
DMDRP Applications Being Accepted
Applications to the Fiscal Year 2013 (FY13) Duchenne Muscular Dystrophy Research Program (DMDRP) are being solicited for the Assistant Secretary of Defense for Health Affairs, Defense Health Program (DHP), by the U.S. Army Medical Research Acquisitions Activity (USAMRAA). The FY13 appropriation is $3.2 million. The vision of the FY13 DMDRP is to extend and improve the function, quality of life, and lifespan for all individuals diagnosed with Duchenne. As such, the DMDRP is seeking to better inform the development of drugs, devices, and other interventions and promote their effective clinical testing. Learn more.
Parliamentary Duchenne Rare Exon Meeting Report
In Spring of this year, PPMD and other Duchenne stakeholders from around the world, convened a meeting with the UK's Shadow Minister of Health, Andrew Burnham, at Parliament. The purpose was focused on Antisense oligonucleotides (exon skipping) and developing an expedited pathway for all children with 'fixable' mutations. The meeting included particular reference to "rare exons." Read the report produced outlining the discussion and next steps.
This Month in Muscular Dystrophy
Hosted by Kevin Flanigan, MD, of Nationwide Children's, "This Month in Muscular Dystrophy" podcasts highlight the latest in muscular dystrophy and other inherited neuromuscular disease research. This month, Jeff Chamberlain, PhD, McCaw Chair in Muscular Dystrophy and Professor in the departments of neurology, medicine and biochemistry at the University of Washington discusses gene and cell mediated therapies for muscular dystrophy. Download now.
Other Research News
Download Podcasts from PPMD'S Connect Conference
Top 7 Most Researched Rare Diseases: Duchenne Muscular Dystrophy
Sarepta Therapeutics announces plans to submit New Drug Application to FDA for Eteplirsen – A significant step for the Duchenne community
PPMD Launches Duchenne Central App
Visit PPMD's Duchenne Research News Section for the latest Duchenne Headlines
MD-CARE Act Late Summer Push
As we move into late summer, we are getting closer to specific committees taking action on the MD-CARE Act. With 73 cosponsors in the House and 15 in the Senate, we need your help to get the MD-CARE Act moving and up our numbers in both chambers. Specifically, we are targeting states on committees that are next to take action on the bill and could help take the legislation into the next phase. Find out how you can lend your voice in a matter of seconds.
Advocating for Open Access
In 2008, PPMD joined the Genetic Alliance and others in the rare disease community, in support of “open access” – essentially ensuring that all NIH funded researchers make available to the public published peer-reviewed manuscripts and articles on a searchable website like PubMed Central or the National Library of Medicines, making them freely accessible to the public within 12 months of publication. We are thrilled to see the mandate starting to have a large impact, as the NIH reports a huge increase in the number of approved manuscripts this year! Learn more.
It's Time to #TackleDuchenne!
We are just weeks away from the 6th Annual Coach To Cure MD on September 28. With already nearly 500 colleges and universities signed on, we are gearing up for the biggest year in CTC history! All we need now…is you! There are so many easy ways you can join our team and help raise awareness. This is our community's chance to reach millions of spectators over a single weekend. It's your chance to tell your story, to talk about Duchenne, to encourage people to help us make a difference, and ultimately to tackle Duchenne. Visit the Coach To Cure MD Website to find out ways you can be involved. Or contact our Team Captain, Danielle Garrigan. Whether you are a die-hard football fan or not, this is your chance to become part of something meaningful, something on a national scale! Join our team!
Tell Us What You Think
Plans are already underway for the 2014 Connect Conference in Chicago, June 26-29, 2014 at the Chicago Marriott Downtown Magnificent Mile. But we want to make sure that we bring you the best Connect Conference possible. If you attended this year's Connect Conference, please take a couple of minutes to fill out this quick survey and tell us what worked and what we can do better next year!
Run For Our Sons
Celebrate Run For Our Sons' 10th Year at the Most Magical Place on Earth!
Run For Our Sons began with a team of 80 runners at the Walt Disney World Marathon Weekend, and 2014 marks our 10th year running at the most magical place on Earth! Come run with us and celebrate 10 years of going the distance to end Duchenne. Races include a Family Fun Run 5K, the brand new 10K race, a half marathon and a full marathon. For the truly enthusiastic runners there's a Goofy Challenge (the half marathon & full marathon on back-to-back days), or try the new Dopey Challenge – all four races on four consecutive days! Meet up with old friends and make new ones at our team pasta party and Victory Party, and make memories that will last a lifetime. We can't wait to celebrate with you!
We need YOU!
Now recruiting for:
Under Armour Baltimore Running Festival - October 12
Rock ‘n' Roll Denver Marathon & Half Marathon - October 20
ING New York City Marathon - November 3
Naperville Marathon - November 10
Philadelphia Marathon - November 17
Walt Disney World Marathon Weekend - January 9-12, 2014
Chevron Houston Marathon & Aramco Half Marathon - January 19, 2014
Rock 'n' Roll New Orleans Marathon & Half Marathon - February 2, 2014
My Voice: Mitchell Peterson, 17 years old
Saturday, August 17 marks the 15th Annual Mitchell's Run Thru Rockford. And for 15 years, the Peterson family has been raising money and awareness for Duchenne. Mitchell Peterson, 17 years old, has been the inspiration behind this event that continues to grow in numbers and momentum. He refuses to be defined by Duchenne and as he begins his senior year of high school, is already planning for a career after college. The Peterson family teaches us about persistence and dedication, and for that we celebrate Mitchell Peterson as our Feature Voice for August!
Upcoming Events
• August 17: Mitchell's Run thru Rockford
• August 17: RFOS 2013 In Memory of Joshua Arndt 5K
• August 31 – September 1: Disneyland Half Marathon Weekend
• September 2: 5th Annual Labor Day Classic
• September 14: Harvest the Dream Dinner & Auction
• September 14: Sam Strong Run 2 End Duchenne
• September 21: Ducky Derby for Duchenne
• September 28: Garrett's Hero Run Duchenne 5K
• September 28: Miles for Matthew 5K/10K
• September 28: Coach To Cure MD
• September 29: Brogan's Run
Check out our full Calendar and sync it with your personal calendar!
Save the Date!
2014 Connect Conference in Chicago
Date: June 26-29, 2014
Location: Chicago Marriott Downtown Magnificent Mile
PPMD has made transition and transition care for people with Duchenne a major focus of our care agenda. Vice President of Care, Kathi Kinnett discusses the goals of a comprehensive transition program and the pre-conference meeting PPMD held earlier this summer. We will continue to delve into this important subject and address the issues of our aging Duchenne population. Read Kathi's blog.
Top Tips on Moving Out and Becoming a Student
Who better knows about transition issues than people with Duchenne going through major life changes? Disability Horizons contributor and recent speaker at PPMD's pre-conference meeting on Transition, Tayjus Surampudi gives his tips, from the viewpoint of a disabled student, on moving away from home and becoming independent.
Salt, Steroids, and Duchenne
Concern over salt intake and the risk of hypertension in the Duchenne community is rather common – especially when steroids are part of the equation. Kathi Kinnett shares her research into this topic and gives helpful tips for you and your entire family to follow.
Major Changes in Physical Therapy Coverage for Individuals with Duchenne and Becker
Obtaining physical therapy services for individuals with Duchenne and Becker muscular dystrophy, and other conditions for which physical therapy (PT) does not result in improvements in range of motion or function, has been a huge source of frustration for patients, parents, and providers. A recent decision involving the Centers for Medicare and Medicaid (CMS) and addressing coverage for PT services directed at maintaining function and range of motion, rather than improving them, should make this a smoother process. Learn more.
Living Duchenne: Weeding
The battle to free your summer garden of weeds can seem overwhelming and impossible to stay on top of. The battle against Duchenne can feel quite similar. FACES Coordinator (and Connect Conference speaker!) Ivy Scherbarth shares her experiences telling the curious what Duchenne means and how to gauge your audience. Read Ivy's latest blog.
More Education Updates
PPMD celebrates the 23rd anniversary of ADA! Read this great blog by Dr. Coleen Boyle, PhD, MShyg, Director, CDC's National Center on Birth Defects and Developmental Disabilities.
Read Mary-Lou Weisman's blog capturing her Connect Conference speech in Baltimore!
PPMD Launches New “Care for Duchenne” Section
Make it Possible!
PPMD'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.

Our family-centered approach is at the heart of everything we do. As your participation grows, so does our ability to strategically invest and navigate in the fight to end Duchenne. Find out how you can become a more active member of this community. Learn more.
Our mission:
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
401 Hackensack Avenue, 9th Floor
Hackensack, NJ 07601
t. 800-714-5437
Parent Project Muscular Dystrophy
PPMD Community
Run For Our Sons
Coach to Cure MD
Facebook Twitter RSS Feed Youtube
© Parent Project Muscular Dystrophy 2013 — All Rights Reserved.

Help us end Duchenne. Donate today.
Forward to a friend. Was this e-mail forwarded to you? Subscribe today.

Unsubscribe from receiving email, or change your email preferences.

Unsubscribe from receiving email, or change your email preferences.