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Go for the Gold
August 2012
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As you read this newsletter, the 2012 Summer Olympics in London have come to a close. The world’s best athletes are heading home with their medals and their memories. For this community, we will remember an Olympian who swam for our sons and daughters who can’t. Ryan Lochte captured Olympic gold (and silver and bronze) and our hearts. And while you cheered him on, you gave towards to potential therapies that PPMD feels strongly about – tamoxifen and SERCA2a. You too, are a champion of Olympic proportions in our eyes! Speaking of champions, we are excited to introduce you to our Featured Voice for August, Sean Quigg the founder of Spaghetti Arms. This is a wildly creative young man who is raising awareness across the country at rock concerts and art festivals. Little does he realize, he’s the real rock star! Finally, as many of you get back into the swing of school and the rest of you prepare for that inevitable first day, we want to update you on the latest care recommendations, give you access to presentations from our 2012 Connect Conference, and make sure you save the date for our upcoming webinar on school issues. Plus, you have to check out the incredible work of photographer Rick Giudotti and Positive Exposure.

Summer’s winding down, but it’s not over yet. Stay cool, keep your head up, and thanks again for supporting our Go for the Gold Campaign!

Pat Furlong, PPMD Founder & President
P.S. Find out where PPMD has been & what we've been doing!


Sean Quigg, 18 years old
Research
Pat Furlong named one of the PharmaVOICE 100
Congratulations to PPMD President & Founder Pat Furlong for being named one of the PharmaVOICE 100! This is an incredible honor and we are so proud of Pat for being recognized as an inspiring part of the healthcare industry and one of the biggest forces in the global Duchenne community. Thanks to PTC Therapeutics for nominating Pat. Read the interview.
Interim data suggests significant clinical benefit with eteplirsen
Sarepta Therapeutics (previously known as AVI) announced a couple of weeks ago that 36 weeks of treatment with its exon-skipping compound, eteplirsen, achieved a significant clinical benefit on the primary clinical outcome, the 6-minute walk test, over a placebo/delayed treatment cohort in a Phase IIb trial in Duchenne patients. The final results of this 48-week study should be available in the fall. PPMD is proud to be an early supporter of exon skipping and Sarepta. Sharon Hesterlee, Senior Research Director of PPMD, explains not only what success in skipping exon 51 means, but how having an effective drug for Duchenne could have widespread benefits, even for those without an exon 51 skippable mutation. Read Sharon's blog.
PPMD Study on Expectations for Clinical Trials
PPMD is conducting a study to understand how the Duchenne and Becker community think and feel about potential therapeutics and clinical trials. The project will focus on motivations, hopes, expectations, and responsibilities. PPMD’s Vice President of Education and Outreach, Holly Peay helps explain why your feedback is critical to this process. Read Holly’s blog.
Sildenafil Findings
A new publication from PPMD grantee and Scientific Advisory Committee member Dr. Stanley Froehner, of the University of Washington, Seattle, demonstrates that phosphodiesterase inhibitor drugs like (sildenafil) Viagra® and (tadalafil) Cialis® can significantly reduce weakness and scarring of the diaphragm muscles in mice that lack dystrophin. PPMD is currently funding a clinical trial to study the effects of Viagra and Cialis in boys between the ages of 7 and 15 at Cedars Sinai Hospital, Los Angeles. See here for more information: Sildenafil and Tadalafil.
Stem Cell Update
The New Directions in Skeletal Muscle Biology and Disease meeting was held June 17-21, 2012 in New Orleans. PPMD sponsored a workshop on stem cell therapeutics. Read notes from that meeting compiled by Dr. Annemieke Aartsma-Rus.
Other Research News
Request for Applications for the next competitive round of the Paul D. Wellstone Muscular Dystrophy Cooperative Research Centers
Progress on Duchenne muscular dystrophy via antisense exon region skipping
Advocacy
Senate and House Continue to Send Message Against CDC Consolidation
Thank you to everyone who reached out to their Senate and House members about stopping consolidation of vital Duchenne programs at the CDC. Members of both the House and the Senate sent a strong message to Dr. Friedan on why they believe Congress spoke clearly in the FY 2012 Consolidated Appropriations Bill. Your continued work is having a tremendous impact. Learn more.
PPMD Endorses The National Pediatric Research Network Act
In a letter to Senators Brown and Wicker, PPMD officially endorsed the National Pediatric Research Network Act legislation that seeks to accelerate the development of new treatments and therapies to combat devastating childhood diseases through a greater investment in basic, translational, and clinical research. The National Pediatric Research Network Act will strengthen and expand pediatric research conducted by the National Institutes of Health (NIH) by establishing national Pediatric Research Consortia (PRCs). This legislation is critical for advancing children's health because it will strengthen the federal investment in pediatric research by authorizing adequate funding for infrastructure and core services needed to support pediatric translational and clinical research for diseases like Duchenne. Learn more.
Advocacy Dates to Remember
Save the date: 2013 Advocacy Conference to be held February 10-12, 2013 in Washington, D.C.
Community
Go for the Gold!
Olympic hero Ryan Lochte will return to the U.S. with five medals – an incredible feat for one of the most talented athletes on the planet. And, as you know by now, he was swimming on our behalf. After losing a relative to Duchenne, Ryan has dedicated his swimming career to raising awareness and helping us raise money for research. Our Go for the Gold campaign has been a huge success with over $200,000 raised for tamoxifen and SERCA2a – therapies that could potentially help all people with Duchenne. We are 2/3 of the way there, so please take a moment and help us reach our goal of $300,000. And encourage your friends to donate or text (JEAH to 90999 to make a $5 donation)! Together we can bring home the gold. Together we can end Duchenne! Make a donation.
Coach To Cure MD: 9.29.12
On September 29, 2011, coaches from your favorite university will be wearing Coach To Cure MD patches for the 5th year in a row to raise awareness for the fight to end Duchenne. To date, Coach To Cure MD has raised $1 million towards the fight to end Duchenne. Will you join our team? Participating in Coach To Cure MD has never been easier. Just text, talk, and tailgate!
Run For Our Sons – Newsletter/Updates
The past six months have been action-packed for Run For Our Sons, and we wanted to take a moment to share what's happened and what's ahead with the entire Parent Project Muscular Dystrophy community. A big thank you to each and every one of you who have gone the distance to end Duchenne, whether it was a full marathon or a 5K - you're helping us raise funds to support PPMD's research, advocacy, care, and education initiatives. Enjoy our updates, and check out our fall and winter race calendar to find your next challenge!
My Voice: Sean Quigg, 18 years old
Creativity, humor, edgy, important – these are just some of the words we can use to describe our Featured Voice for August, Sean Quigg and his Spaghetti Arms project. Sean wanted to raise awareness in a clever way that explained Duchenne. He also wanted an excuse to follow some of his favorite bands. Thus, Spaghetti Arms was born, whose mission is to raise awareness of Duchenne through music and art events. Or as Sean puts it – making the curious aware. Whether hosting events, manning booths at some of the summer’s biggest musical festivals, selling shirts, or giving away stickers, Sean and his friends are educating their peers, opening eyes, and changing minds. Sean is an open and honest entrepreneur and artist, quickly becoming a prominent voice of his generation. Meet Sean Quigg.
Upcoming Events
• August 11: Volley for Mitchell
• August 18: Mitchell's Run thru Rockford
• August 18: RFOS 2012 IMO Joshua Arndt 5K
• August 18: Last Splash for Duchenne- Canada
• September 1-2: Disneyland Half Marathon & 5K
• September 2: 4th Annual Labor Day Classic
• 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 our full Calendar and sync it with your personal calendar!
Education
Annual Connect Conference
PRESENTATIONS NOW AVAILABLE
We are proud to share presentations from this year’s Annual Connect Conference in Ft. Lauderdale. The conference was packed with great information, and now you can access anything you might have missed and want to learn more about. Also, make sure you download the care sheets (adapted from the CDC Care Considerations guidelines published in Lancet in 2009) and Endocrine sheets that are a result of a PPMD sponsored Endocrine workshop.
POSITIVE EXPOSURE
If you were with us in Florida, you probably saw the beautiful portraits of mothers and sons taken by our incredible key note speaker Rick Guidotti. Learn more about Rick’s Positive Exposure project and his work with people with Duchenne.
MORE CONFERENCE UPDATES
Save the date: For next year’s Connect Conference as we return to Baltimore, June 27-30.
Let us know how we did: 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!
Back to School Webinar
The next webinar in our Direct Access series will be Wednesday, August 29 at 1pm eastern. We will be discussing IEPs and assistive technology as our kids head back to school. Moderators Brian Denger and Jill Castle will go over what you need to know when talking to your child’s school. Topics will include: The Individualized Education Program (IEP) meeting – what to expect, your role and how to deal with conflict and challenges, as well as support services commonly provided for children with Duchenne. The webinar will also cover Assistive technology and how that fits into an IEP. Learn more.
Standardizing Duchenne Care
The clear message received by all at the end of the Transforming Duchenne Care meeting, held just prior to the PPMD Connect Conference, was that parents, providers, and industry felt a need for clear, consistent standards of care for patients with Duchenne around the United States. Read the latest update by Kathi Kinnet about PPMD’s ongoing efforts to help standardize Duchenne care.
Two Moms, Co-Blogging: Perspective
Parents agonize over clinical trials, balancing the pros and cons and above all else, wanting to do what is best for not just the physical health of their child, but the emotional health as well. In this month’s co-blog, Colorado FACES coordinator and parent Ivy Scherbarth and PPMD's Pat Furlong share their own conversations with their sons about clinical trials and where priorities sometimes lie. We hope you will share the conversations you’ve had with your kids, after you read these blogs.
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.
Coach To Cure MD
On September 29, 2011, coaches from your favorite university will be wearing Coach To Cure MD patches for the 5th year in a row to raise awareness for the fight to end Duchenne. To date, Coach To Cure MD has raised $1 million towards the fight to end Duchenne. Will you join our team? Participating in Coach To Cure MD has never been easier: Just text, talk, and tailgate!
Shop For Our Sons

It’s that back to school time of year! And as you get the kids ready to head back, don’t forget to turn to Amazon for all of your back to school shopping – not just books! When you shop Amazon, using our link, you help raise money for research! Visit ShopForOurSons.org and click the Amazon link. Then shop away. It’s that easy! A portion of all of your purchases will go to PPMD.
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
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© Parent Project Muscular Dystrophy 2012 — All Rights Reserved.

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