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FDA Continues to Hear Us
July 2013
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First things first: Thank you!

PPMD's Annual Connect Conference in Baltimore was a huge success and I'm extremely grateful to everyone who made the trek from all over the world to be with us. To connect. Families, extended families, researchers, care providers, scientists, physicians, pharmaceutical representatives, members of government agencies – all gathered to discuss progress and promise. We are so grateful to the presenters who took time out of their schedule to be with us and share with us. I know many of you were unable to attend for a variety of reasons. We are excited to share downloadable podcasts from many of the powerful and informative presentations given.

While Baltimore was great with lots of information shared, lots learned – there is still more work to do. Just last week, several of us from PPMD were off to Washington, D.C. Some attended a Congressional briefing on Capitol Hill to urge attending staffers to support the reauthorization of the MD-CARE Act and to show their support for accelerated approval. At the same time, myself and a group of leaders in Duchenne research, as well as parents, met with top officials at the FDA to discuss accelerated approval, what we have learned about the natural history of the disease, and the importance of incorporating benefit risk considerations into the review process.

Washington continues to listen. Progress continues to be made. Our voices, united, will end Duchenne!

I also want to take a moment to introduce you to our Featured Voice for July, John Owen Dumm. He and his family's foundation, John Owen's Adventure, Inc., continue to fundraise for this community and just recently supported PPMD's cardiac initiative. Meet this great 9-year old and his family!

It's a hot, hot summer and I hope you and your family are staying cool and enjoying some quality time together.


Pat Furlong
PPMD Founder & President


Research
Downloadable Podcasts from PPMD's Connect Conference
If you missed this year's Annual Connect Conference or if you attended and want to revisit a favorite presentation, we are happy to provide downloadable podcasts. A lot of information was shared in a short amount of time and it can be overwhelming. We want to give you the opportunity to listen and re-listen to important information that was shared. Continue to check back on our landing pages as more presentations will be posted as they become available.
Clinical Trials Update
A staple of each Connect Conference has become our conference guide featuring clinical trial updates for all Duchenne and Becker related trials, from those currently recruiting to pre-clinical, and everything in between. Special thanks to Ann Martin, DuchenneConnect Coordinator and Genetic Counselor for compiling this great resource. Download the clinical trial sheets today.
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. Paul T. Martin, principal investigator, Center for Gene Therapy and Neurosciences Center, The Research Institute at Nationwide Children's Hospital discusses how overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice. Download now.
Duchenne Research News in One Place
Don't forget to find more of the latest research news on PPMD's homepage under Duchenne Research News and powered by Scoop.it! Follow us, so that you can get updates the minute we post them. Or, visit our Duchenne Research News section periodically and catch up on what you might have missed.
Other Research News
Congratulations to PTC Therapeutics on announcing their initial public offering. This makes over half a dozen publically traded companies working in Duchenne research!
PPMD Launches Duchenne Central App
Visit PPMD's Duchenne Research News Section for the latest Duchenne headlines
Advocacy
July 9, 2013 – Duchenne Takes Over D.C.
Tuesday, July 9 was a big day for Duchenne advocacy. Advocates spoke about the high unmet medical need and urgency for treatments, with meetings at the FDA and a Congressional briefing. PPMD President Pat Furlong and Director of Advocacy Ryan Fischer provide updates on both these meetings, including responses from agency leaders and next steps. We are strongest when we speak in one voice and with one mission: End Duchenne.

PPMD Presents to EMA
Late last month, PPMD President Pat Furlong, along with Elizabeth Vroom from Duchenne Parent Project in the Netherlands and Filippo Buccella from Parent Project Onlus in Italy, presented to the European Medicines Agency (EMA) in response to their draft guidance on clinical studies in Duchenne. The EMA is the European equivalent of the FDA here in the U.S. We are proud to represent the Duchenne community and will keep you posted on any outcome! You can read the presentation Pat delivered on the PPMD blog.
We Were Heard
Your advocacy matters! Duchenne was included in a group of eight diseases areas for a new pilot program to discover new therapeutic uses for existing molecules launched by the National Center for Advancing Translational Sciences (NCATS), with financial support from the NIH Common Fund. When you lend your voice, Washington hears you!
More Advocay Updates
PPMD Urges HHS Secretary to Update Organ Transplantation Policies to Ensure Pediatric Patients Have Access
Community
Thank You!
Thank you to everyone who joined us in Baltimore for the 2013 PPMD's Annual Connect Conference! While the extensive agenda, research presentations, break-out sessions, and interactions with industry leaders were all an incredible part of this year's conference, the highlight for the PPMD staff was being able to connect and reconnect with all of you – our awe inspiring community.

We are a community of fiercely passionate people. And at PPMD we are confident that we – you, us – will continue to make the impossible, possible. Together, we will end Duchenne.
Official Photos from Connect Conference
Our wonderful photographer, Jeff Mauritzen from inPhotograph has provided a link of the amazing pictures he captured at this year's conference. If you are interested in copies of any of these photos, contact Jeff through the link provided. We think you'll agree, he really captured the spirit of our Connect Conference!
9.28.13 – Coach To Cure VI

Is your team on our team? On September 28, 2013, Coach To Cure MD returns for a sixth year! We already over 340 colleges and universities registered to participate, so 2013 is well on its way to being our biggest year yet! You may be the person, though, to help convince your favorite university to join Coach To Cure MD. Click here to see if they are registered. If not, contact our team captain Danielle Garrigan.

Many universities have also expressed an interest in having families attend their game day. Would you like to participate? Fill out our survey and we will do our best to get you there!

Coach To Cure VI – Let's #TackleDuchenne!

Run For Our Sons
Runner Spotlight
This month's runner spotlight is on NJ couple Mike & Amy Napoli, long-time RFOS team members who will be running the Inaugural Dopey Challenge at the Walt Disney World Marathon Weekend in January 2014 in honor of their nephew Danny. Meet Mike & Amy.
Race Recap
A big thank you to our dedicated team of 25 runners at the Rock 'n' Roll Seattle Marathon & Half Marathon, including our roller, Tatsuro Nakajima, who competed in the half marathon in his power wheelchair. Our Seattle-area families continue to make this one of our most successful races of the summer. Thank you to all of our runners and those who support them for going the distance to end Duchenne!
We need YOU!
Now recruiting for:
Medtronic Twin Cities Marathon - October 6
Under Armour Baltimore Running Festival - October 12
Rock ‘n' Roll Denver Marathon & Half Marathon - October 20
Marine Corps Marathon - October 27
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: John Owen Dumm, 9 years old
For six years now, the Dumm family in North Royalton, Ohio has been hosting JOA Picnic in the Park to raise money and awareness for Duchenne research. This is just one of the many things John Owen's Adventure, Inc. (JOA) does for our community. At the heart of JOA is 9-year-old John Owen Dumm who is a rising fourth grader with Duchenne and an active participant in his namesake event. In fact, John Owen and JOA have been so successful in their fundraising efforts, they recently granted PPMD a $20,000 gift for our cardiac initiative. This month we celebrate the kid behind a great foundation that seeks to help all people with Duchenne! Meet our Feature Voice for July, John Owen Dumm!
Upcoming Events
• July 27: Friar Fight to End Duchenne Golf Tournament
• August 2: Ballin' for Billy
• August 3: Fabian's Rally of Hope to End Duchenne
• August 3: MRC Day 5K and Family Fun Run
• 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 28: Coach To Cure MD
Check out our full Calendar and sync it with your personal calendar!
Education
PPMD Receives World Orphan Drug Congress Honor
PPMD is proud to be named one of the top 50 social media influencers in orphan drugs and rare disease by the World Orphan Drug Congress. We strive to be your go-to resource for Duchenne news and updates, and we hope you'll continue to follow us on Facebook, Twitter, YouTube, and Scoop.it!
Answering Your Duchenne Care Questions
PPMD Vice President of Care, Kathi Kinnett helps answer a common Duchenne respiratory question regarding the best support for your child's pulmonary issues: the cough assist vs. "the vest." Kathi turned to Drs. Richard Shell (Nationwide Children's Hospital, Columbus, Ohio), Hemant Sawnani (Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio), and Jonathan Finder (Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania) for their expert opinions. Read her findings.

Kathi also addressed the very serious concern of use of anesthesia in dental procedures. Turning to a team of experts, Kathi has reported what she learned about local anesthetic and nitrous oxide combined with Duchenne.
Living Duchenne: Talking About Duchenne
How do you talk to your child/children, as well as others, about Duchenne? There's no one easy answer. But 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.
Weisman Gifts
PPMD was doubly blessed at this year's Connect Conference to receive two incredible gifts from the Weisman family. First, Mary-Lou Weisman – a favorite author and blogger of this community – gave a fantastic closing dinner speech to an enthralled audience, sharing her experiences, her story with Duchenne. Then, her husband Larry joined her onstage to present Pat with a $100,000 gift from the Peter B. Weisman Fellowship. The Weismans have supported the work of rising researchers with fellowships in the past. Thank you hardly seem adequate to the continuing generosity of this wonderful family!

Make sure you check out Mary-Lou's blog, including her latest story about having Liza Minnelli play her!
More Education Updates
PPMD Published Article: Transforming Duchenne Care
PPMD Launches New "Care for Duchenne" Section
DuchenneConnect Presents 2012 Data & 2013 Update
Download PPMD's updated emergency care card featuring revised information on giving oxygen to people with Duchenne.
Make it Possible!
PPMD's mission is to end Duchenne. 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. Learn more about how we help.
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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