Parent Project Muscular Dystrophy
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Better, Faster, NOW
October 2012
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Wow! What an unbelievable few weeks we have had in this community! Never in the 18 year history of PPMD has the Duchenne community been so ripe with promise and so full of hope. As I’m sure everyone is aware, Sarepta Therapeutics presented positive results from their 48-week results on eteplirsen, a drug designed to skip exon 51. PPMD will host a webinar for you to ask your questions on October 23. Just last week, Summit announced that they have been able to achieve sufficient plasma levels of its reformulated utrophin-upregulating drug to justify a phase I/II trial in Duchenne. This is a therapy that would potentially help all people with Duchenne. PPMD has contributed to both therapies over the years and is excited for each company’s next steps. We continue to fund important research by awarding grants to Dr. Dongsheng Duan for his gene therapy work in treating cardiomyopathy in Duchenne and Dr. Toshifumi Yokota at the University of Alberta who is experimenting with multiple exon skipping. This month’s World Muscle Society Congress in Perth, Australia promises one exciting announcement after another.

I also want you to meet a really cool guy: Wheelz. Wheelz (a/k/a Ryan Schmidt) is a rapper who, along with his family, hosts a huge Duchenne-awareness event in Austin every year. He uses music to enlighten audiences about Duchenne and is a force to be reckoned with. Meet Wheelz and make sure to download some of his music!

You will see that this newsletter is packed with information concerning advocacy, care, additional research news, incredible webinars, and the many goings-on in the community. It is a time like no other in this community and I hope, like PPMD, you will face the end of the year full of hope and optimism.

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


Ryan "Wheelz" Schmidt, 20 years old
Research
PPMD Awards Over $300,000 in Grants
PPMD is proud to award research grants to Dr. Dongsheng Duan for his gene therapy work in treating cardiomyopathy in Duchenne and Dr. Toshifumi Yokota at the University of Alberta who is experimenting with multiple exon skipping. Both projects use innovative science that if successful, will extend the quality of life of people with Duchenne. We are so grateful to have this kind of talent working for our community.
Positive Phase I trial results for SMT C1100 (Summit PLC)
Summit PLC announced that they have been able to achieve sufficient plasma levels of its reformulated utrophin-upregulating drug to justify a phase I/II trial in Duchenne. (Read Summit's Press Release). This is promising news for a therapy PPMD has supported along with other significant funders in the Duchenne community. Learn more about what these results mean from PPMD’s Senior Director of Research, Dr. Sharon Hesterlee. And visit Summit's new website.
Promising News from Sarepta on Exon 51 Skipping Trial
The much-anticipated 48-week results on eteplirsen, a drug designed to skip exon 51, were presented today by Sarepta Therapeutics on a webinar and press release. On the webinar, Sarepta’s Chief Executive Chris Garabedian presented data about the dystrophin found in study participants’ muscle biopsies and the 6-minute walk results. PPMD’s Vice President of Education and Outreach, Holly Peay, helps to shed light on the results.
PPMD’s Sharon Hesterlee Featured in Nature Magazine
In an article published in the most recent issue of Nature magazine called, "A call for transparent reporting to optimize the predictive value of preclinical research," PPMD’s Senior Director of Research, Dr. Sharon Hesterlee helps take a look at reasons why animal data was not translating efficiently into good drug candidates for various diseases. In her accompanying blog, she explains PPMD’s Better, Faster, Now philosophy as it relates to current research in the Duchenne space.
Upcoming Webinars
PPMD will be hosting two important webinars in the coming weeks. Please mark your calendars and make sure to join us. As is usually the case, if you are unable to attend or if you want to take another listen, links to the webinars will be provided.
Q&A WITH SAREPTA
This is your opportunity to find out more about the 48-week results of eteplirsen and to ask your questions. Learn more.
MD-STARNET
On our Oct 31 webinar, learn about MD STARnet, a program that aims to better understand the Duchenne and Becker community: our numbers, our medical service use, and our needs. Dr. Dennis Matthews will present "MD STARnet Revealed."
Other Research News
Prosensa Named a Fierce 15 Biotech Company - 'One of the most promising private biotechnology companies in the industry'
Advocacy
PPMD President receives RARE Champion Award in Advocacy
On September 27, rare disease patients, advocates, patient organizations, government agencies, pharmaceutical companies, medical researchers, celebrities, Olympic champions, and private sector representatives attended the Global Genes | R.A.R.E. Project 1st Annual "RARE Tribute to Champions of Hope" benefit. PPMD President Pat Furlong was honored to receive the RARE Champion Award in Advocacy. Read her blog about this magical evening.
Understanding Accelerated Approval
With the recent news of Sarepta's promising 48-week data, many have asked how accelerated approval may play into the FDA review of eteplirsen. PPMD’s Director of Advocacy, Ryan Fischer, explains what accelerated approval means and how it can play into the advancement of a drug like eteplirsen.
Implementing PDUFA
With Congress decamping from Washington and not expected to return until after the November election, we thought we'd revisit one of the few bright spots of bipartisanship this Congress – the enactment of the latest FDA user fee law (PDUFA), known as the FDA Safety and Innovation Act or FDASIA. Read Ryan Fischer’s latest blog explaining what’s next with PDUFA.
Advocacy Dates to Remember
Save the Date: 2013 Advocacy Conference - February 10-12, 2013 in Washington, D.C.
Community
End Duchenne Gala - 12.6.12
Please join us for the inaugural End Duchenne Gala on December 6, 2012 from 7:00 pm - 12:00 am at the Metropolitan Club in New York City, NY. The End Duchenne Gala will celebrate current research advancements and help raise funds for upcoming research that will help us change the future for those living with Duchenne. This promises to be a truly special night and we hope you will join us. Learn more.
Run For Our Sons
As the temperature starts to drop, it is perfect weather to start running! Check out what Run For Our Sons has going on near you.
WALT DISNEY WORLD MARATHON WEEKEND
(JANUARY 10-13, 2013)
Our entries for the Walt Disney World Marathon Weekend are filling up quickly! We have half marathon and 5K entries left. Don’t miss out on one of our most fun races of the year! Visit our website to register today.
WE NEED YOU!
Now recruiting for other winter races as well:
Chevron Houston Marathon - January 13, 2013
ING Miami Marathon & Half Marathon - January 27, 2013
Rock 'n' Roll New Orleans Marathon & Half Marathon - February 24, 2013
RFOS TEAM CONQUERS THE WINDY CITY
Twenty five runners conquered 26.2 miles through Chicago in one of the world's most prestigious marathons, and raised nearly $40,000 in the process! Check out the full recap.
HOW I BECAME A RUNNER
PPMD’s Vice President of Development, Mary Connolly blogs about getting her start as a runner.
Coach To Cure MD V: Another Huge Hit!
On September 29, over 10,000 coaches from around the country on over 500 college teams wore Coach To Cure MD patches in honor of the fifth anniversary of this national Duchenne-awareness event. From press conferences to coin tosses to radio interviews and sport commentator updates, our message to end Duchenne was shared with one of the largest audiences this community has access to. Events will continue over the coming weeks, patches will keep being worn, and donations will keep coming in. As soon as we have results of what was raised, we will let you know. But until then, thank you for continuing to support Coach To Cure MD and we hope you’ll join us next year as this program continues to grow!
My Voice: Ryan "Wheelz" Schmidt, 20 years old
Wheelz (a/k/a Ryan Schmidt) is a 20-year-old musician from Austin, Texas. He is an accomplished rapper, and with his family, hosts Ryan’s Day – one of the largest annual fundraising and awareness events for Duchenne in Austin, held last week. The combination of his inspirational raps and hosting Ryan’s Day remind people outside the community that we are in a fight to end Duchenne and need every bit of support we can get. It’s for this reason—his dedication to raising awareness and inspiring people through his craft—that we choose Wheelz as our Featured Voice for October. Meet Ryan “Wheelz” Schmidt.
Upcoming Events
• October 20: Harvesting Hope for Andrew 5K Run/Walk
• October 21: Metro Health Grand Rapids Marathon
• October 21: Rock 'n' Roll St. Louis Marathon & Half Marathon
• October 23: Sherlock’s On the Green Golf Tournament
• October 25: End Duchenne Pizza-Thon
• October 27: Sam’s Day
• October 27: Brookhaven 5K Run/Walk
• October 27: 1st Annual Moonachie Mystery Run
• Novemebr 4: ING New York City Marathon
• November 10: Grand Rapids .1K
• November 17: Gunnar Listerman Memorial 5K Run, Walk, & Roll for Duchenne
Check out our full Calendar and sync it with your personal calendar!
More Community News
PPMD named one of NerdWallet’s Top 6 Healthcare Innovation Nonprofits
HerSelf First Fall Newsletter
Education
PPMD Cardiac Webinar Series: Interventions, 11.28.12
The next webinar in PPMD’s new cardiac care series will be on November 28 at 1pm eastern. The topic of this discussion will be interventions. Dr. John Lynn Jeffries from Cincinnati Children’s Hospital and Dr. Larry Markham from Children’s Hospital Vanderbilt will be our guests. If you missed our first webinar last month regarding monitoring heart disease in Duchenne and carrier moms, click here to watch the full presentation and to find answers to questions you might have asked. Learn more about the upcoming webinars.
Calling All Mothers: We Need Your Input!
Parent Project Muscular Dystrophy’s Women’s Project is still in need of moms to participate in the Mothers’ Survey. This survey collects information from mothers of children with Duchenne and Becker, including your needs, your strengths, and your wellbeing. The results from this survey will help us develop new interventions to improve mothers’ wellbeing.
HerSelf First Pod Cast: Intimacy & Desire
For many caregivers, maintaining healthy, intimate connections with their partners becomes challenging in the wake of busy, complicated lives. Renowned speaker, author, sexuality counselor, and certified nurse mid-wife Evelyn Resh joins the conversation to offer insight into why intimate relationships are a matter of good, health practices that ultimately benefit the entire family unit and not simply privileged "indulgences." Download and listen.
Two Moms, Co-Blogging: Virtual Moment of Silence
This month, instead of a pair of essays, we are calling for a Virtual Moment of Silence. We invite you, our Duchenne/Becker Muscular Dystrophy community, to use the comments area to list the names of the loved ones you remember and miss. We honor all who have been touched by Duchenne and Becker muscular dystrophy: the brave men and boys, women and girls who lived with Duchenne all their lives; the carrier mothers and sisters; the kind doctors who made a real difference; the beloved service dog who served faithfully until the end.

Rest in Peace. You are our heroes and we celebrate your spirit.
Other Education Updates
Management of End Stage Heart Failure: Ventricular Assist Devices (VAD)
Protecting Family Caregivers from Employment Discrimination.
Medical Alert Bracelets for Duchenne
The Flu Vaccine & Duchenne: What You Need to Know
Are You Ready? Preparedness Tips for People with Mobility Disabilities
Save the Date: PPMD’s West Coast Connect Meeting, April 26-28, 2013 in San Diego
Make it Happen!
End Duchenne Gala
Parent Project Muscular Dystrophy will honor heroes in the fight to end Duchenne at our first End Duchenne Gala on Thursday, December 6, 2012 in New York City.

Mark your calendars and plan to 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.
Shop For Our Sons
Need a last minute Halloween costume ASAP? Starting your holiday shopping early? In charge of Thanksgiving this year and need pots and pans? Amazon has everything you can imagine. When you shop Amazon, using our link, we 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. Happy Fall!
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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