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Time to Unite & Rally
November 2013
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The news from Sarepta this week was devastating. The news from GSK and Prosensa a few weeks back was devastating. But as I said in my blog about this recent news, now is not the time to turn on each other or give in to the negativity. PPMD will keep working for you and with you so that the incredible momentum we have had going in 2013, continues.

Next up – and truthfully the timing couldn’t be more perfect – is the first-ever Duchenne Policy Forum on December 12. You can learn more about the agenda and goals of this meeting by clicking here. You can also participate in this forum by either attending in person, or sharing your story, your frustrations with the FDA.

Joining us at the Policy Forum will be Ansel Lurio, a 29-year-old museum education major and outspoken advocate for Duchenne. Ansel will share his experience living with Duchenne and we couldn’t be more grateful to have him, not only as one of our panelist, but also as our Featured Voice for November.

I know that there is not a lot of comfort that can be offered at a time when bad news seems like it continues to plague this community. But I will say – remember you are not alone. You have your PPMD family who is here to support you and who will continue to work to end Duchenne, even in moments when you may not have the strength.

Warm regards,

Pat Furlong
PPMD Founder & President

Facing Disappointment from FDA's Eteplirsen Response
By now many of you may have heard the news that the FDA has called the new drug application for the exon 51 skipping drug eteplirsen premature. They have requested instead that a larger phase III study be conducted before they will consider the application for approval. PPMD, which provided travel support to families who participated in the trial, is deeply disappointed in this decision, but hopeful that the FDA will provide clear guidance in the near future on the quantity and quality of data that is required for it to consider an accelerated approval or an application based on phase II data. Although accelerated approval and phase II approvals are possible, they are not common. Read more. And read PPMD President Pat Furlong’s latest blog, Time to Unite & Rally.
PPMD & Sarepta Decode Duchenne
We are excited to announce that Parent Project Muscular Dystrophy and Sarepta Therapeutics are joining forces on a genetic testing program for patients with Duchenne or Becker muscular dystrophy. Through the new genetic testing program, called Decode Duchenne, PPMD will provide testing at no cost to eligible patients who are unable to access testing due to barriers such as a lack of or insufficient insurance coverage. Sarepta is supporting the PPMD-administered initiative. Learn more about Decode Duchenne.
Decode Duchenne Nominated for ROAR Award
We're excited to see so many Duchenne-related initiatives nominated for Rare & Orphan Advocacy and Research (ROAR) Awards this year including the just-launched Decode Duchenne! Decode Duchenne was nominated for "Best European Industry-Patient Organization Engagement." Prosensa and CureDuchenne's work was also recognized, as well as the work of other pharmaceuticals and biotechs with Duchenne drug development programs.
Who Owns Your Clinical Trial Data?
What happens when the volumes of data that are generated from a clinical trial remain in the hands of a single company or academic investigator… sometimes for years while additional analysis is completed and publications are drafted and submitted? Even when immediate release of the data could help avoid duplicative research, improve safety in future trials, or decrease the time it takes to conduct additional trials?

PPMD's Vice President of Research Sharon Hesterlee was asked to speak at the Institute of Medicine Committee’s first public meeting on the topic of data sharing in clinical trials, and discussed the urgency to work out the details, because real people who count time in weeks and minutes are being affected. Read more.
Other Research News
Ataluren Trial Now Recruiting at All US Sites
Summit receives regulatory approval to start Phase 1B Clinical Trial of SMT C1100
Prosensa has announced that they have successfully enrolled the 100th patient into their Natural History Study of Duchenne.
Let's Skip Ahead: This new resource from Sarepta Therapeutics offers an exon mapping tool that allows visitors to explore the potential link between specific genetic mutations in Duchenne and exon skipping.
Register for DuchenneConnect and stay connected to the latest updates on clinical trial recruitment.
PPMD Announces Landmark Duchenne Policy Forum
PPMD is proud to host a Policy Forum on clinical trials of experiment agents on December 12, 2013 from 8.30AM until 4.30PM in Silver Springs, MD. We are especially pleased that key Duchenne-community stakeholders, including the U.S. Food and Drug Administration (FDA), Center for Drug Evaluation and Research (CDER), and review divisions, are committed to the success of this day-long meeting. This meeting will be chaired by two well-regarded experts, Dr. Craig McDonald and Dr. Lee Sweeney.

"Optimizing Clinical Trials in Duchenne Muscular Dystrophy in the New Era of Improved Care Standards" is designed to create a draft guidance for the FDA that reflects the combined experience and wisdom of the Duchenne community. To find out how you can lend your voice to the process and participate visit the event page.
Action Alert! The MD-CARE Act
We are still in the process of gathering cosponsors for the legislation. Ryan Fischer, PPMD’s Director of Advocacy and Outreach just published a blog on the current status of the bill for the community at large. And while we are making great progress, there is still much more work to be done! In order to move the bill through Congress, we need to up our numbers. Now is the time to reach back out to your representatives on Capitol Hill and ask for their support. And if you have already reached out, recruit your friends and family who might be asking what they can do to help us end Duchenne. This is a simple action anyone can take! Read Ryan’s blog to learn more.
Help Update the MD Action Plan
Over the summer the Muscular Dystrophy Coordinating Committee (MDCC) met to discuss the next update to the MD Action Plan. As legislated by the MD-CARE Act, the MDCC is tasked with creating the Action Plan for the Muscular Dystrophies and also with periodically updating it to reflect the current landscape. Brian Denger currently sits on the committee representing the Duchenne community and PPMD, and recently reported back on the August meeting. Please check out Brian's recent blog and read about how you can help inform the update.
Save the Date!
PPMD's Annual Advocacy Conference
Date: February 23-25, 2014
Location: Marriott Wardman Park
Save the Date
Other Advocacy News
Read last month’s interview with PPMD President Pat Furlong regarding PPMD's recently published white paper on benefit/risk
PPMD Shares White Paper with FDA: Benefit-Risk Assessments in Rare Disorders
Pat Furlong Receives Lifetime Achievement Award
Because of changes in health information resulting from the Affordable Care Act, the National Organization for Rare Disorders has provide a State-by-State Insurance Information webpage helping you to determine changes that may affect you and your family.
Run For Our Sons
Run For Our Sons 10th Year Celebration
The registration deadline has been extended for our 10th Year celebration at the Walt Disney World Marathon Weekend in January! Join us for this very special weekend that includes a special surprise gift for each of our runners, and create family memories that will last a lifetime. Races include the inaugural Dopey Challenge and a new 10K, and old favorites including the half and full marathons. Register today for the most magical place on Earth!
Runner in the Spotlight
This month's runner spotlight is on Shane Lauer, a 21-year-old man living with Duchenne who recently participated in his fifth half-Ironman triathlon! This is his incredible story.
Fall Race Recaps
From the Inner Harbor of Baltimore to the streets of the Windy City, our runners and walkers completed 5Ks, half marathons, and full marathons with passion, perseverance, and more than a few smiles! Check out the recaps of all of our fall races, and thank you to all of our Run For Our Sons teammates for going great distances to end Duchenne this year.
We need YOU!
Now recruiting for:
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
Rite-Aid Cleveland Marathon - May 18, 2014
My Voice: Ansel Lurio, 29 years old
In a few weeks, PPMD will be hosting a Policy Forum on clinical trials of experiment agents This is a landmark meeting that will include participation from the FDA. It will also include Ansel Lurio, our Featured Voice for November. Ansel is a 29-year-old with Duchenne and has a great deal of experience sharing his story with decision makers and industry professionals. His charisma, his eloquence, and his willingness to talk about his experiences, we believe, will help guide the FDA and will be a great representation of the Duchenne patient community. Meet our Featured Voice for November, Ansel Lurio.
Upcoming Events
• November 16: Richmond Marathon
• November 17: Philadelphia Marathon
• November 23: Gunnar Listerman Memorial 5K
• November 27: Hustle 4 Muscle
• December 6: Ekdor 2013 Golf Tournament
• December 6: Hope for the Holidays Dinner & Auction
• January 9-12, 2014: Walt Disney World Marathon Weekend
• January 19, 2014: Chevron Houston Marathon & Aramco Houston Half Marathon
• February 2, 2014: Rock 'n' Roll New Orleans Marathon & Half Marathon
• June 21, 2014: Rock 'n' Roll Seattle Marathon & Half Marathon
Check out our full Calendar and sync it with your personal calendar!
Save the Date!
2014 Connect Conference in Chicago
Date: June 26-29, 2014
Location: Chicago Marriott Downtown Magnificent Mile
Save the Date
The ‘JB's Keys to DMD’ Scholarship Fund at Northeastern University
JB’s Keys to DMD will fund a scholarship, available starting in the fall of 2014, providing a full scholarship for a qualified applicant living with Duchenne to attend Northeastern University. The person must have been accepted to Northeastern University through the typical application process and deemed qualified by Northeastern's Disability Resource Center. The scholarship will include all academic, room, and board expenses as well as stipends for medical support while on campus or while on co-op. To learn more, click here.
What Do You Think About Clinical Trials?
Though clinical trials aren’t for everyone, each trial helps move us toward better treatments. More and more, companies and clinicians are asking parents for their thoughts and feelings – and they are coming to PPMD as a resource. What do you think about clinical trials? Share your thoughts, so PPMD can give the best advice to professionals running trials. Click here to learn more.
Inside a Case Study for Pediatric Heart Failure
PPMD's Vice President of Clinical Care, Kathi Kinnett, goes deep inside a case study for pediatric heart failure. Learn more from this important cardiac summit Kathi just attended.
Important Flu Vaccination Updates for 2013-14
Flu season is back and the vaccine is slightly different this year. Nasal spray flu vaccines are not recommended. Injected flu shots are recommended for patients with neuromuscular diseases and patients taking chronic steroids. Learn more.
Living Duchenne: Identification
"I have rarely been comfortable at the center of anyone's attention.Yet one of the most profound changes wrought on my life by Duchenne has been a tendency to be noticeable. People see me and my family now in a way that I had not experienced before. Strangers offer their visual disapproval at every turn." Ivy Scherbarth talks about the public pressure to be a good Duchenne role model in her latest blog. Let us know about your experiences dealing with a society that so often doesn't 'get it.' Check out Ivy's blog.
More Education Updates
Updates from the Child Neurology Society Annual Meeting in Austin, TX
Make it Possible!
Leveraging the United Way
The annual United Way campaigns are getting underway throughout the country. Last year many families had great success leveraging both the annual United Way campaign and Combined Federal Campaign (CFC) to raise money to fight Duchenne. Many people have a tradition of donating to the United Way, are expecting their company campaigns to start, and are looking for good causes to earmark their donations. View PPMD's United Way/CFC Fundraising Campaigns guide (PDF) which provides information on how local United Way operate and how to work to arrange funding through them.
Shop For Our Sons
With Thanksgiving comes the beginning of holiday shopping, including the two biggest sales of the year: Black Friday (11/29) and Cyber Monday (12/2). Avoid the crowds and enjoy your family by shopping from home through Amazon. When you shop Amazon, using our link, we raise money for research! Visit and click the Amazon link. It’s that easy! A portion of all of your purchases will go to PPMD.

And print out this flyer to give to friends, family, your child's school, or wherever you can think of. It's a simple way people can shop and make a difference!
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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