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Research & Advocacy Efforts Will #TackleDuchenne
September 2013
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Slowly the leaves are turning, the kids are back at school, maybe the lightweight coats are coming out of storage. Fall is upon us and Coach To Cure MD is just days away! I love this time of year because in many ways, it's a new start – everyone is in game mode, recharged and ready to tackle the issues at hand. For this community, the issue is singular and always the same: end Duchenne.

We continue to be fortunate enough to have the ongoing support of the AFCA and nearly 600 universities around the country for the 6th Annual Coach To Cure MD on September 28, 2013. On this day, every college game on every network will be wearing out patches. This is an incredible awareness opportunity for the general public and a great fundraiser. Over $1 million has been raised for research since the program's inception. Even if football isn't your thing, on September 28 it is your chance to make it your thing: tell your story, host an event, text a donation... get in the game!

The success of Coach To Cure MD is because of families like TJ Clime's. TJ is a 9 year old from Jefferson City, Missouri. He and his family have supported Coach To Cure for the last several years by encouraging their local high school to participate. The result – TJ led the team on the field in one of their first games! This great third grader is a champion and a fighter and our Featured Voice for September. Meet TJ Clime.

It's with the precision, determination, and strength of a seasoned and trained athlete that this community continues to fight Duchenne. I was privileged to speak on behalf of Duchenne and rare diseases at recent meetings hosted by NINDS and FDA. We are being heard and I left encouraged. More Members of Congress are signing on to the Reauthorization of the MD-CARE Act. Our Google alerts continue to be filled with research updates and clinical trial announcements.

Events like Coach To Cure MD, Run For Our Sons, all of the amazing grassroots fundraisers that happen across the country – these events inform, open hearts, and bring us that much closer to the end zone.

Thank you for being on our team and be proud on September 28 when you see the Coach To Cure logo!


Pat Furlong
PPMD Founder & President


Research
Webinar Replay: Prosensa Update on Duchenne Program
Dr. Giles Campion, Chief Medical Officer at Prosensa presented information about plans for development of follow-on molecules to drisapersen in Duchenne. If you missed this webinar or want to revisit any of the presentation, click here.
Explanation: Duchenne Transcript Determines Dystrophin Levels
A recent article published in The FASEB discussed new observations that the size of the messenger RNAs produced from dystrophin genes with mutations is often much shorter than a normal length messenger RNA. This finding may have implications for the efficiency of exon skipping. Annemieke Aartsma-Rus, PhD, has provided insight to help clarify the data discussed in this article.
Notes from the 2013 MDCC Meeting
Late last month, several members of the PPMD team attended the Muscular Dystrophy Coordinating Committee (MDCC) meeting in Washington, D.C. The MD-CARE Act authorized the establishment of the MDCC to coordinate activities across NIH and with other Federal health programs and activities relevant to the various forms of muscular dystrophy. Click here for extensive notes from PPMD's Parent Advocate, Brian Denger.
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, Dr. Volker Straub discusses the use of muscle MRI in muscular dystrophy. Dr. Straub is the Harold Macmillan Professor of Medicine and Professor of neuromuscular genetics at the University of Newcastle upon Tyne in the United Kingdom. Download now.
Other Research News
PPMD Funds $1 Million in Exon Skipping Projects
Download Podcasts from PPMD's Connect Conference
PPMD Launches Duchenne Central App
Advocacy
PPMD Presents at Two Influential Meetings
We are honored both NINDS and FDA have recognized our work to ensure the patient and parent perspective – particularly on critically important topics such as benefit/risk, trial design, and clinical and surrogate endpoints – is listened to. PPMD President Pat Furlong was asked to present at meetings last week and provides an update.
State By State Insurance Information
In anticipation of changes in health information resulting from the Affordable Care Act, the National Organization for Rare Disorders has provide a State-by-State Insurance Information webpage we think will be helpful in determining changes that may affect you and your family. Click here to find resources to answer your questions about your health insurance plan.
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 74 cosponsors in the House and 16 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!
Community
#TackleDuchenne: 9.28.13!
We are just days away from the 6th Annual Coach To Cure MD on September 28. With almost 600 universities signed on – our biggest enrollment ever! – we are gearing up for an event that we believe will make Duchenne history! There is still time for you to get involved, join our team, and make this year's event even stronger. 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!
Back To School
Thank you to all of our families who shared their back to school photos. We hope everyone has a wonderful, new school year!
Run For Our Sons
Run With Us in Baltimore!
The Baltimore Running Fesitval has quickly become one of our largest races, with nearly 80 runners of all ages participating in the half marathon, full marathon relay, and 5K race. Join in the fun on Saturday, October 12th, and celebrate at our after-party that evening! Last year we raised more than $120,000 and we need YOU to help us raise even more this year!
Our Newest Addition
Run For Our Sons is proud to add the Philadelphia Marathon to this year's line-up of races. Join us on November 17, 2013 in the City of Brotherly Love for what is consistently rated one of the top 10 races in the country. This year marks the race's 20th anniversary, making it an extra special year to run!
Runner in the Spotlight
This month's spotlight is on Todd Burns, who competed in the 2013 Ironman 70.3 World Championship on September 8th in Las Vegas in honor of family friend Sam Killian. Meet Todd.
A Magical Weekend at Disneyland
A huge thank you to our 110 runners who ran the inaugural Dumbo Double Dare, Half Marathon, 10K, and 5K races at the Disneyland Half Marathon Weekend, raising more than $115,000 to help us end Duchenne! These incredible runners endured a heat wave over Labor Day Weekend and finished the races with smiles on their faces. A special congratulations to Diane Fuller, who came in first in her age group in both the Half Marathon and the 10K!
We need YOU!
Now recruiting for:
Under Armour Baltimore Running Festival - October 12
Rock ‘n' Roll Denver Marathon & Half Marathon - October 20
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: TJ Clime, 9 years old
On September 28, 2013, families across the country will celebrate PPMD's Sixth Annual Coach To Cure MD. Hundreds of high school coaches (and entire teams!) will support this amazing event. TJ Clime and his family suited up and started supporting Coach To Cure MD weeks ago. This incredible third grader and his family have been on the field and TJ ran with the team to break through the sign to start a recent game! Through fundraising and telling their story to raise awareness, TJ and his family are some of the MVPs of this year's Coach To Cure MD. So meet our Featured Voice for September, TJ Clime!
Upcoming Events
• 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
• October 5: Rosstoberfest
• October 5: Shea Holbrook Duchenne Racing Helmet Raffle
• October 5: Sam's Day
• October 6: 2013 Twin Cities Marathon
• October 12: Baltimore Running Festival
• October 12: Argyle Run For Our Sons 5K/1Mile Fun Run
• October 13: Bank of America Chicago Marathon
• October 20: Rock ‘n' Roll Denver Marathon & Half Marathon
• October 26: Brookhaven Family Run
• October 27: 2013 Marine Corps Marathon
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
More Community Updates
3 Moms, 1 Mission: See how Mitchell's Run Thru Rockford affects parents of local boys
Spiritual Journey to the Arches: Check out an amazing blog in photos by PPMD FACES of Arizona Coordinator Jill Anne Castle, mom to Oliver, 11, and Anthony, 13 and living with Duchenne.
Education
Talking To Teachers
Meeting with your child's teachers and key staff is one of the most important things you can do for your child to be confident when sending him off to the first day of class. Check out a list of resources we've compiled for parents and teachers to help bolster that confidence. What are some of your concerns for the new school year? What resources would you add? Join the discussion!
Ensuring Equal Employment
Vice President Biden announced recently a new rule that represents a historic advance for individuals with disabilities. By strengthening longstanding regulations under Section 503 of the Rehabilitation Act, the new rules will ensure that qualified workers have more meaningful opportunities to find, secure, and keep good jobs.
Living Duchenne: How I Wish
Have you ever daydreamed about what the perfect visit to the doctor(s) would be like for you and your child with Duchenne? FACES Coordinator (and Connect Conference speaker!) Ivy Scherbarth shares her imaginary visit in her latest blog. Check out Ivy's blog and share with us what you're perfect scenario would include.
Fighting Duchenne
For the last few years, PPMD has reached out to photographer Jeff Mauritzen to capture images from some of our most important events. Jeff has been deeply moved by the people in this community that he has photographed – so much so that he put together a blog to share his experience. Check out how Jeff has captured just a few of the great kids in this community.
More Education Updates
Salt, Steroids, and Duchenne
Major Changes in Physical Therapy Coverage for Individuals with Duchenne and Becker
Make it Possible!
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.
CONTACT
Parent Project Muscular Dystrophy
401 Hackensack Avenue, 9th Floor
Hackensack, NJ 07601
t. 800-714-5437
SITES
Parent Project Muscular Dystrophy
PPMD Community
DuchenneConnect
Run For Our Sons
Coach to Cure MD
CONNECT
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© Parent Project Muscular Dystrophy 2013 — All Rights Reserved.

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