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A New Year, A New Message for Congress
January 2013
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Though we’re already a couple of weeks into 2013, Happy New Year! I hope your holidays were wonderful and that you are looking towards this year as one of promise in our community. Last year was like no other in the history of Duchenne. After years of feeling like nothing was happening, 2012 brought a boom of research. And that momentum is continuing. We’ve already run marathons at Walt Disney World and in Houston, we met our holiday fundraising goals, and we have awarded grants to critical research projects.

Next on our agenda is something that we need your help with: the Reauthorization of the MD-CARE Act. We hope you will join us in Washington, D.C. from February 10-12 as we head to Capitol Hill and share our stories with new members and returning members. If you can’t join us, we hope you’ll participate from home through one of several ways. The important thing is to include your voice. The MD-CARE Act has been the single greatest legislation in the fight to end Duchenne, leveraging hundreds of millions of dollars for research and care. We can’t let this funding disappear. The lives of our children depend on us being their voices and sharing their stories.

Speaking of voices in this community, one of the most optimistic people with Duchenne you will ever meet is this month’s Featured Voice, Anthony DeVergillo. He is a charismatic young man who is using his journey with Duchenne to inspire others to reexamine their life and find the silver lining.

PPMD is looking at this new year as a year of not just doing something, but changing something. Everyone has a talent or passion they can share that will lead us that much closer to ending Duchenne. We hope you will look at what we are doing across our variety of programs and activities, and figure out how you can help make the impossible, possible.

Happy 2013!

Pat Furlong
PPMD Founder & President

You made it possible!
Thanks to your support this holiday season, we met the full $50,000 match and reached our ambitious goal of $300,000. We're thrilled with the hope that this accomplishment brings—hope that an end to Duchenne is more possible than ever. We will fund projects that will provide important outcome measures for non-ambulatory as well as very young persons with Duchenne, setting the stage for the significant number of clinical trials coming forward. Read Pat’s letter and then watch a special thank you message.
Minidystrophin gene therapy effective in dogs
Dr. Dongsheng Duan of the University of Missouri has reported strength improvements in dogs that lack dystrophin after injecting a modified virus carrying a new design of a miniaturized dystrophin gene into the dogs’ muscles. The new “minidystrophin” contains a functional region of the gene that has not been used in previous version of minidystrophins used in human and dog gene therapy studies. In parallel, PPMD has supported Dr. Duan’s work to develop an effective gene therapy approach for treating the heart—his publication on the optimum virus subtype to use in the heart is in progress now and stayed tuned as well for reports on his progress in using the gene for a molecule called SERCA2A to treat cardiac problems in Duchenne. Learn more about Dr. Duan’s grants: SERCA2a gene therapy for Duchenne cardiomyopathy and Identify novel AAV vectors for canine heart transduction by peripheral vein injection.
Cancer drug Tamoxifen may prove useful for Duchenne
A report out today in the American Journal of Pathology by Dr. Olivier Dorchies and Urs Ruegg of the University of Geneva demonstrates that mice without dystrophin that were treated with the breast cancer drug Tamoxifen developed normal strength and showed a 50% reduction in fibrosis, or scarring, in their muscles. PPMD is co-funding, along with United Parent Project MD, studies necessary to verify these initial results and lay the groundwork for a human clinical trial. This project is part of PPMD’s “Now” strategy to test approved drugs.
GSK on the Progress of Drisapersen
The last patient has been recruited within the drisapersen US Phase II clinical study. Drisapersen is GSK’s exon 51-skipping drug. This exploratory study aims to assess the safety, efficacy, and pharmacokinetics of two doses of drisapersen in the treatment of ambulant boys with Duchenne who have a dystrophin gene mutation amenable to an exon 51 skip. The study is expected to complete in November 2013, with study results available in early 2014. Learn more.
ENMC Workshop Update
The 194th workshop of the European Neuro Muscular Centre (ENMC) met in Naarden, The Netherlands, December 7-9, 2012. Read workshop reports from the ENMC on newborn screening and exon skipping. Plus revisit the recap blog from PPMD’s Director of Care, Kathi Kinnett who attended and reported on what took place, including in-depth discussions on the future of exon skipping.
Webinar Reminder: PTC to Provide Ataluren Update,
1.17.13 at noon eastern
Dr. Jay Barth, Vice President for Clinical Development at PTC Therapeutics will present information about the study design and timeline for the upcoming Phase 3 confirmatory study of ataluren in Duchenne on Thursday, January 17 at noon eastern. Learn more.
Other Research News
Vice President of Research, Sharon Hesterlee, Ph.D., co-presented PPMD’s unique method of evaluating projects via the TREAT-NMD TACT committee at the Faster Cures Partnering for Cures meeting
You & the Reauthorization of the MD-CARE Act
This year's annual Advocacy Conference is one of the most critical events on PPMD's 2013 calendar. And we need you! We face challenging times in our nation’s capitol. With talk of the debt ceiling and other fiscal matters filling the halls of the capitol building we MUST keep Duchenne on Congress’s priority list and continue the momentum we started over 10 years ago. We cannot afford to lose the gains we’ve made since the first MD-CARE Act in 2001. Will you join us in February to make the case to members of Congress about why we must pass this bill for the third time? We must do it for the sake of all those living with Duchenne, and for all those diagnosed tomorrow and beyond.

The conference will take place February 10, 11, and 12. Click here to register before the February 1 cut off.
Can’t attend?
Send in your information for our coordinated packet drop off and do a follow up phone call while we are on the Hill.
Some exciting news to report!
Ryan Lochte will be joining us this year in DC! This will be a great press opportunity and will help bring attention to the need for reauthorization. Ryan will be in attendance at training Sunday, visiting offices on Monday, and at our reception on Monday night.

Please contact Ryan Fischer if you have any questions about the conference. Reauthorization of the MD-CARE Act is possible. We just need your voice!
Everyone Can Advocate
One of the easiest ways you can make a difference in the fight to end Duchenne, is advocacy. The word can sound intimidating or complicated, but it’s not. Check out our GET ACTIVE IN ADVOCACY landing page and find out how you can be involved.
More Advocacy News
Last Chance to Register: 2013 Advocacy Conference to be held February 10-12, 2013 in Washington, D.C. Registration closes February 1!
Your Go-To Duchenne News Resource
You’re stressed, you’re overwhelmed, you’re bombarded with news/statuses/tweets/texts…and the last thing you have time for is deciphering what is good information and what is a waste of your time and energy. That’s why at PPMD, we are doing our best to bring you at least the Duchenne news and information you need. It’s a noisy online world and we want you to feel like you can turn to PPMD and our various outlets to get thoughtful, vetted information when you need it. There is enough to overwhelm you and your family when Duchenne comes into your life. We hope you will look to PPMD as a guide, as a friend, as a group you can trust to find answers and make connections. Find and follow us.
Dreams Can Come True!
Many of you saw on Facebook, the story about a young man with Duchenne who had a holiday wish. Thanks to the power of social media and everyone who shared his story, 14-year-old Blake Dudley realized his dream of seeing The Fighting Irish play for the National Championship in person! A donation from American Airlines and an anonymous donation of three game tickets, helped Blake's dream become a reality! Read Blake's story.
Conquer the Canyon to End Duchenne
Last year, 12 brave individuals conquered the Grand Canyon in honor and in memory of very special people in their lives who were affected by Duchenne. PPMD and Conquer The Canyon have joined forces again to bring you Conquer The Canyon to End Duchenne, a once in a lifetime, 3-night/4-day walking, hiking and active adventure at one of the "Grandest" natural wonders of the world - The Grand Canyon! Get ready for a very rare experience. Less than 1% of the millions of visitors to the park each year ever travel its trails and with Conquer The Canyon, you have the opportunity to join this exclusive group.

From May 3-6, 2013 PPMD is inviting you to join us on this exclusive, once in a lifetime adventure to help us end Duchenne. We've made it simple for you - let us arrange for your transportation, lodging, some meals and park admission. All you need to do is arrive in Phoenix ready for the adventure of your life! We have wheelchair-accessible hikes available, so bring the whole family.

Visit our website for an itinerary and more information.

We hope you'll join us on this brand new adventure into one of the natural wonders of the world!
Run For Our Sons
Recap: Walt Disney World Marathon Weekend
170 runners ranging in age from 5 to 67 celebrated the Walt Disney World Marathon’s 20th anniversary January 10th-13th. This fun-filled weekend included a 5K, half marathon, full marathon and Goofy Challenge, team pasta and victory parties featuring Phinneus & Ferb. And most importantly, our team raised more than $260,000 to help us end Duchenne. Thank you to each and every runner you are the heroes of our community!
Recap: Chevron Houston Marathon
Rain and freezing temperatures couldn’t stop our 100 runners who participated in this year’s Chevron Houston Marathon, Aramco Houston Half Marathon and ABB 5K races. They endured the bad weather with smiles and raised more than $95,000 to support PPMD’s work. Thank you to the Save Our Boy Foundation for sponsoring our team and to all of the runners who made this a memorable weekend for the Duchenne community.
Aquaphor New York City Triathlon
Run For Our Sons becomes Tri For Our Sons for the Aquaphor New York City Triathlon! If you’re looking for a new adventure, this iconic event could be for you. The Olympic-distance triathlon takes you on a swimming, biking, and running adventure through the Big Apple, before you cross the finish line in New York’s famed Central Park. Join us on Sunday, July 14 and go the triathlon distance to end Duchenne.
We need YOU!
Now recruiting for:
Rock 'n' Roll New Orleans Marathon & Half Marathon - February 24
Conquer the Canyon to End Duchenne - May 3-6
Cincinnati Flying Pig Marathon - May 5
Rite-Aid Cleveland Marathon - May 19
Rock 'n' Roll Seattle Marathon & Half Marathon - June 22
Aquaphor New York City Triathlon - July 14
Disneyland Half Marathon, 10K & Family Fun Run 5K - August 31 & September 1
Bank of America Chicago Marathon - October 13
My Voice: Anthony DeVergillo , 19 years old
When you meet Anthony DeVergillo in person, you can’t help but leave the conversation with a smile on your face. This almost 20 year old is the definition of optimism. In fact, he has made spreading optimism his life’s work. From an early age, Anthony realized he had two choices with his Duchenne diagnosis let it defeat him both physically and emotionally, or challenge it head on with a great attitude and the power of positive thinking. Through his blog, Anthony is reaching members of this community and helping them turn their lives around by simply adjusting their attitude. That’s why we are thrilled to introduce to you our Featured Voice for January, Anthony DeVergillo.
American Taxpayer Relief Act of 2012
On January 1, 2013, Congress enacted the American Taxpayer Relief Act of 2012 into law. The bill affects charitable giving in many ways and we felt it was important to share this information with you. Read PPMD’s Vice President of Development, Mary Connolly’s latest blog about what this means for you and your charitable giving in 2013.
Upcoming Events
• January 27: ING Miami Marathon & Half Marathon
• February 9: Bingo Battle to End Duchenne
• February 9: Game, Set, Cure MD
• February 10-12: Advocacy Conference
• February 23: UF Physical Therapy PPMD Charity Golf Tournament
• February 24: Rock 'n' Roll New Orleans Marathon & Half Marathon
Check out our full Calendar and sync it with your personal calendar!
PPMD Cardiac Webinar Series: The Use of
Ventricular Assist Devices (VAD) in Duchenne, 1.24.13
The next webinar in PPMD’s new cardiac care series will be on January 24, 2013 at 1pm eastern. This is a hot topic in the community. The discussion will include: when a VAD might be considered; what is a VAD/LVAD; types of VADs; challenges of choosing a device; patient selection; and the pros/cons of VAD implantation. For more background information on VADs in Duchenne, read PPMD’s Director of Care, Kathi Kinnett’s latest blog.

We have also schedule an additional webinar for March 6, 2013 at 2pm eastern. The discussion will focus on care in adults living with Duchenne.

If you missed last month’s webinar on cardiac interventions, click here to download. It includes answers to questions posed during and after the webinar.
Educating healthcare providers about the need for early diagnosis
Families of children with muscular dystrophy and other muscle disorders often experience a diagnostic odyssey that is too long and too emotionally difficult. PPMD, with support from The Centers for Disease Control and Prevention (CDC), created the National Task Force for Early Identification of Childhood Neuromuscular Disorders to address the continued gap between the time when the first symptoms of muscle weakness are noticed and the time of a neuromuscular diagnosis. Learn more about this important task force.
Living Duchenne: My Year in Review
Each month, Colorado FACES coordinator and parent Ivy Scherbarth will you bring you her thoughts related to the Duchenne journey. This month, Ivy summarizes 2012 a year that held significant moments for her and her family, not just because of Duchenne. There is a universality to her specific story and we hope it inspires you to share some of your thoughts on your own journey last year. Read Ivy’s latest blog.
Other Education Updates
Save the Date: PPMD’s West Coast Connect Meeting, April 26-28, 2013 in San Diego
Save the Date: PPMD’s Connect Conference, June 27-30, 2013 in Baltimore
Make it Possible!
Run For Our Sons
In 2005 PPMD hatched a plan to recruit 20 runners for the Walt Disney WorldŽ Marathon. We wound up with a remarkable 86-member team, which raised $186,000 for Duchenne research. Since then, Run For Our Sons has blossomed into an extraordinary community fundraising program, drawing thousands of individuals and families and raising over $6 million. And along the way, they have a great time doing it! To learn more visit
Share Your Memories
Are you hosting an event for PPMD? Running a race for Run For Our Sons? Have you partied for Coach To Cure MD? Or do you have a stack of great photos from your experiences at the conference lying around? We need your photos and videos! Anything you have that reflects you and your family working to end Duchenne we need. Next year marks PPMD’s 20th anniversary and we want to mark the occasion through your eyes. Share with us today!
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
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