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The Theater of Duchenne
July 2012
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I think at this point we all get that ending Duchenne is going to take a collaboration of efforts. Treatments will come from different researchers, produced by different pharmaceutical companies, and supported by different foundations. I wrote an essay entitled The Theater of Duchenne that was included in the 2012 PPMD Connect Conference User's Guide (contact us if you're interested in a copy of the guide), in which I compared treating Duchenne and the clinical trial process to producing a Broadway hit. I wanted to put an often confusing process down on paper to help even me better wrap my head around the players and policies involved in treating Duchenne. It's just one of the many things we shared at arguably our most comprehensive and informative conference to date. You'll find several highlights in this month's newsletter with presentations coming soon. We heard from lots of amazing people at the Conference, and wanted to highlight two voices that stood out. Jack and Nolan Willis are 10 year old twins with Duchenne who participated in one our panel about clinical trials from the patients perspective. Their insight, experience, and humor helped shed light on what patients experience when they are in trials.

The heat is on around the country - so stay cool and if you are on summer vacation, enjoy!

Pat Furlong, PPMD Founder & President
P.S. Check out how I've been spending my summer by reading my latest recap of where we've been and what we've been doing!


Jack and Nolan Willis, 10 years old
Research
Ataluren Update
In their latest update, PTC summarizes ataluren's efficacy and safety as we now understand it, extension studies now being opened outside the US, their regulatory path forward, and the potential for a confirmatory study. Read the update (PDF).
PPMD's Pat Furlong Featured in Boston Globe Article
PPMD President Pat Furlong was recently featured in a Boston Globe article about the power of the patient voice in driving new drug research. It's a fascinating look at the evolution of patient advocacy and drug discovery.
Prosensa wins "Emerging Star Award" at the European Mediscience Awards
Congratulations to Prosensa, the Dutch biopharmaceutical company focusing on rare diseases with a high unmet medical need (including and perhaps especially Duchenne) for winning the Emerging Star accolade at European Mediscience Awards. Read the release.
Other Research News
AVI BioPharma changes its name to Sarepta Therapeutics
Clinical trial first to test heart drug regimen for Duchenne muscular dystrophy
Advocacy
Action Alert! Contact your Senators to help save vital Duchenne programs at the CDC
The deadline has been extended and we need your voice to help save critical Duchenne related programs at the CDC, now more than ever! Efforts to consolidate these activities threaten to slow down the gains we have made in treating Duchenne. In May, you contacted your member in the House of Representatives. Now it's time to contact your two Senators! Deadline: Friday, July 20. Learn more & take action.
Landmark FDA legislation becomes law
PPMD applauds the President for signing into law the Prescription Drug User Fee Act (PDUFA) also known as The Food and Drug Administration Safety and Innovation Act. This is a major win for the Duchenne community, as well as, the rare disease community as a whole. We are grateful to everyone who helped by contacting your members of Congress to educate them on the importance of the provisions we championed. Your voice made all the difference! Read PPMD's Director of Advocacy, Ryan Fischer's latest blog for more.
Community
Go for the Gold!
At PPMD's Annual Connect Conference, we received a wonderful video message from Olympic medalist and world record holder Ryan Lochte. Ryan has been a longtime supporter of this community and a champion of our sons. His message challenges all of us to "Go for the Gold" and raise money for specific research projects so that we can be victorious too, and get that much closer to ending Duchenne.

To date, we have raised $189,120 of our $300,000 to fund SERCA2a and tamoxifen. We hope that as you watch Ryan win more medals and break more world records in the coming weeks, you will join him in his support of PPMD by giving to our Go for the Gold campaign and funding this critical research that we believe will help all of our sons dream Olympic dreams, and one day see those dreams come true.
How to Leverage the United Way to End Duchenne
Bob and Ruth Getler have updated their guide to help parents and friends of people living with Duchenne to better leverage the established framework of the United Way, the Federal Workers Combined Federal Campaign (CFC), and other charity campaigns to raise funds in support of ending Duchenne. Developing a better understanding of each type of fundraising campaign will enhance and accelerate your overall fundraising efforts. Download this helpful guide.
Run For Our Sons
RECENT RACES
We teamed up with AVI to put together an amazing team for last month's Rock 'n' Roll Seattle Marathon & Half Marathon, including "roller" Conrad Reynoldson, who completed his second half marathon in his power wheelchair! Read PPMD's Endurance Program Manager, Nicole Herring's blog about Conrad and the AVI BioPharma partnership. And thank you to the entire Run For Our Sons Seattle team for raising nearly $25,000 to help us end Duchenne.
Congrats and thank you to our first ever Tri For Our Sons team! These 15 men and women completed an Olympic-distance triathlon on a hot July day and raised more than $40,000! Check out the team photo and race recap. If you're interested in being part of the 2013 Tri For Our Sons team, email us at info@runforoursons.org. Entries will be available in the fall and will go quickly!
UPCOMING RACES
Have you signed up for a fall or winter race yet? Now's the time to choose your race and hit the ground running (literally!) with your training. Here are some great upcoming races across the country:
Rock 'n' Roll Philadelphia Half Marathon - September 16
2012 Baltimore Running Festival - October 13
Rock 'n' Roll St. Louis Marathon & Half Marathon - October 21
2012 Grand Rapids Marathon - October 21
ING New York City Marathon - November 4
Walt Disney World Marathon Weekend - January 10-13, 2013
Chevron Houston Marathon - January 13, 2013
Rock 'n' Roll New Orleans Marathon & Half Marathon - February 24, 2013
CONQUER THE CANYON - NOW RECRUITING!
We're recruiting adventurous hikers for our 2013 Conquer the Canyon to End Duchenne trip! There are accessible Canyon trails as well so bring the whole family for a once-in-a-lifetime experience you will never forget. And check out this video from our 2012 trip.
Pat Furlong Featured in Thrive! Magazine
Duchenne advocate and parent Misty VanderWeele of Alaska, has launched a magazine called Thrive!, geared towards Duchenne families. This month's cover is our own Pat Furlong. Read the interview with Pat and check out Misty's website.
My Voice: Jack and Nolan Willis, 10 years old
The clinical trial process is complicated and confusing. At this year's Annual Connect Conference, PPMD wanted to shed light on what the experience was like for parents, clinicians, industry, and most importantly, patients. For our final panel, we assembled four young men who have had experiences in different trials to talk about what it was like. Included in this amazing discussion were Jack and Nolan Willis, 10-year-old twins from Fayetteville, New York. They were candid, honest, and funny. By sharing their insight, the audience better understood what the clinical trial experience is like for younger patients and how selfless these kids are in participating on behalf of all of our sons. Meet Jack and Nolan.
Upcoming Events
• July 18-22: Spaghetti Arms at Warped Tour
• July 27-28: JOA Picnic in the Park (PDF)
• July 28: Friar Fight to End Duchenne Golf Tournament
• August 18: Mitchell's Run thru Rockford
• August 18: RFOS 2012 IMO Joshua Arndt 5K
• September 1-2: Disneyland Half Marathon & 5K
• September 2: 4th Annual Labor Day Classic
• September 8: Ducky Derby
• September 15: Miles for Matthew
• September 16: Comic Relief
• September 16: Rock 'n' Roll Philadelphia Half Marathon
• September 22: Rock 'n' Roll Denver Marathon & Half Marathon
• September 29: Coach To Cure MD
Check out the Calendar and sync it with your personal online calendar!
More Community Updates
Are you following the amazing Tim Rice on his thousand mile hike along the Pacific Crest Trail on behalf of PPMD? Read the latest as he continues to take step after step to end Duchenne!
Education
Summary from PPMD's 'Transforming Duchenne Care' Meeting
One of the highlights of this year's Annual Connect Conference was the Transforming Duchenne Care meeting which took place just before the general sessions got underway. The response to our invitation to join this first-of-its-kind meeting, was overwhelming. Pat Furlong and Director of Care, Kathi Kinnett, have compiled a summary from the meeting. Read Kathi's blog to learn more and to find out about next steps.
Highlights from PPMD's Annual Connect Conference
NEW RESOURCES
As mentioned in Pat's opening note, this year's Annual Connect Conference in Ft. Lauderdale was incredibly informative, comprehensive, and included the leaders of the Duchenne fight. In the coming weeks, we will be providing links to presentations (both PowerPoint slides and downloadable podcasts, when possible) from the Conference. In the meantime, we wanted to share some of the great resources that made their 'debut' this year.
CLOSING NIGHT DINNER
Another highlight of the Conference was Luca Buccella's moving speech at the closing night dinner on Saturday. Son of Filippo Buccella, the President of Parent Project Italy and a friend of PPMD's for years, Luca inspired us and made us laugh. Watch his speech and read a transcript.
MORE CONFERENCE HIGHLIGHTS
• Check out the PPMD Connect Conference landing page in the coming weeks for presentations as they are posted.
• If you attended this year's Connect Conference, we would love to know what your experience was like and how we could improve. Fill out this quick evaluation and let us know how we did!
SAVE THE DATE for next year's Connect Conference as we return to Baltimore, June 27-30, 2013.
Two Moms, Co-Blogging: Renaissance Thinking
At PPMD's Annual Connect Conference last month, Pat Furlong shared her essay, The Theater of Duchenne. In it, Pat dissects the clinical trial process by drawing comparisons to all of the teamwork that goes into producing a Broadway hit. Colorado FACES coordinator and parent Ivy Scherbarth responds to Pat's essay with her latest blog. We hope you will share your thoughts after you download and read the in-depth essay, The Theater of Duchenne and Ivy's thoughts.
More Education Highlights
Read about the importance of social skills for kids living with Duchenne in the latest article by Jill Castle, a Parent Information Network Specialist for the Arizona Department of Education and the PPMD FACES of Arizona Volunteer Coordinator.
• HerSelf First founder Sheila Moeschen, PhD, was recently featured at MariaShriver.com for her story, Pic Your Path: The Story of Our Steps. Check out this latest project, helping caregivers invest in their own wellbeing.
Make it Happen!
End Duchenne Gala
SAVE THE DATE: Parent Project Muscular Dystrophy will host its first End Duchenne Gala on: Thursday, December 6, 2012 at 7:00 pm - 12:00 am at The Metropolitan Club in New York City, NY. Join us for an incredible evening that will bring together those determined to Change What's Next in the fight to End Duchenne. For more information, visit EndDuchenneGala.org.
Go for the Gold
To date, we have raised $189,120 of our $300,000 to fund SERCA2a and tamoxifen. We hope that as you watch Ryan win more medals and break more world records in the coming weeks, you will join him in his support of PPMD by giving to our Go for the Gold campaign and funding this critical research that we believe will help all of our sons dream Olympic dreams, and one day see those dreams come true.
Shop For Our Sons
The kids are home for the summer but it doesn't mean that learning should stop! Stock up on great books while they are home and do it for a great cause. Visit ShopForOurSons.org and click the Amazon link. Then shop away. It’s that easy! Share this site and encourage your friends to shop for our sons!
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 2012 — All Rights Reserved.

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