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Weathering the Storm
November 2012
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So many of our friends and family, including the New Jersey office of PPMD, were affected by Hurricane Sandy a couple of weeks ago, and then hit again by a huge snow storm. To our knowledge, friends of this community survived, but many went days without power, heat, and water. Some lost a little to flooding or downed trees. Others lost virtually everything. As I watched from overseas and saw the devastation of a part of the country I have come to know as a second home, I saw something else emerge. I saw people helping each other. Elementary schools in states across the country, pulling together supplies for the victims. Families opening their doors to neighbors, friends, even strangers in a time of extreme need. Politicians – politicians! – working together, across the aisle to bring relief. And I thought – this is just like our community. This is what Duchenne families do. We help each other, we reach out to each other, we mend each other. And like Duchenne, help doesn’t often come as fast as we would like or as completely as we would like…but eventually, progress is made, baby step by baby step.

Our community has known and will continue to know setbacks. But we are also starting to learn what victory feels like – no matter how small. You’ll see more research progress in this newsletter as PPMD continues to pursue a mantra of Better, Faster, Now. You’ll see the groundwork we are laying in Washington, D.C., with the FDA with the hopes that promising treatments are understood by federal agencies and accelerated approvals are considered. You’ll see our commitment to care, making sure parents are armed with the tools they need to help their child.

I also want you to take a minute to meet another remarkable person in this community with Duchenne. Michael Counterman is a born entertainer and teacher, who helps people understand Duchenne through laughter. We have all weathered storms – both literally and figuratively. But I hope, like me, you see this community as a refuge and will continue to turn to us in times of need.

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

Research
Duchenne Drug Development Round Table
PPMD held its second "Duchenne Drug Development Round Table" meeting to bring together representatives from different companies working on therapeutics for Duchenne. At the table were also MDA, DART Therapeutics (spun out of Charley’s Fund and the Nash Avery Foundation), and CureDuchenne, and several National Institutes of Health (NIH) representatives. Read Senior Research Director Sharon Hesterlee’s blog about this meeting.
Q&A Webinar with Sarepta
On October 23, PPMD hosted a webinar with Sarepta to discuss the 48 Week dystrophin and 6-minute walk test (6MWT) data from the Phase IIb extension study in Duchenne, as well as, next steps for the eteplirsen program. Chris Garabedian, President and CEO of Sarepta, presented the data and answered submitted questions. View the presentation.
Summit Announces that Repeat Dosing of SMT C1100 Meets Endpoints
SMT C1100 has successfully met the endpoints as part of a Phase I clinical trial in healthy volunteers. The trial evaluated a new formulation of SMT C1100 and the results showed that upon repeat dosing, concentrations of the drug achieved in the blood plasma, stabilized at levels that from preclinical studies are expected to significantly increase utrophin protein production. The new formulation was also shown to be safe and well-tolerated in this Phase I trial. Read the release.
Prosensa Announces Progress on Exon Skipping Compounds
Prosensa has selected clinical candidates for two more compounds for the treatment of Duchenne and has been granted orphan drug designation for two additional Duchenne compounds in its pipeline. Learn more.
GSK Publishes Summary Results Report for Phase I Drisapersen Trial
This Phase I trial involved drisapersen, GSK's exon 51 skipping therapy, formerly known as GSK2402968 and assessed the safety and tolerability of drisapersen after a single subcutaneous injection, at different dose levels, in non-ambulatory boys with Duchenne. There were no serious adverse events reported for any boys in the study. Read the results.
Other Research News
The October 31 webinar with MD-STAR Net was cancelled due to the hurricane. We will reschedule in the coming weeks.
Advocacy
Partnering with the FDA to accelerate approvals
We are at a critical moment in Duchenne history, when it comes to our relationship with the FDA. We continue to partner with the FDA, discussing accelerated approval and risk tolerance. It has never been more important for us to work together as a community for the sake of all people in our lives living with Duchenne. Read Pat’s advocacy update.
FDA’s Patient Focused Drug Development Initiative – Request for Public Comment
PPMD and other organizations recently submitted a joint statement, making the case for neuromuscular disorders affecting children. This letter urges the agency to strongly consider convening a public meeting dedicated exclusively to Degenerative Neuromuscular Disorders Affecting Children as part of the agency's patient-focused drug development initiative. Read more.
Duchenne and the Election
Just because the election has come and gone, doesn’t mean our advocacy work can rest. Now is the time for you to start reaching out to your newly elected candidates or returning officials and build a relationship with them. Learn more.
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
There are still tickets available 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 by visiting EndDuchenneGala.org.

PPMD’s Vice President of Development, Mary Connolly, blogs about her experience putting this year’s Gala together.
Run For Our Sons
Runner Spotlight
This month's runner spotlight is on Leeandra Archdeacon, the Southern California mom of Nicholas. She has participated in nine half marathons and is training for at least four more! Meet Leeandra.
The Show Must Go On!
The 2012 New York City Marathon is the race that wasn't - with Hurricane Sandy clean-up a main priority for the city of New York, the race was cancelled for the first time in its 40+ year history. But many of our runners did not let that stop them! They met in Central Park and ran 26.2 miles anyway, in honor of those they love who are living with Duchenne. Thank you to ALL of our runners who worked so hard over the past few months to raise an amazing amount of money to support PPMD! Race or no race, our entire community is so proud of you!

To check out the rest of our recent race recaps, visit our website.
We need YOU!
Now recruiting for other winter races as well:
Chevron Houston Marathon - January 13
ING Miami Marathon & Half Marathon - January 27
Rock 'n' Roll New Orleans Marathon & Half Marathon - February 24
Conquer the Canyon to End Duchenne - May 3-6
Cincinnati Flying Pig Marathon - May 5
Rite-Aid Cleveland Marathon - May 19
Aquaphor New York City Triathlon - July 14
Free Money!
We hoped that would get your attention! Fall means employee giving campaigns are underway. Make the most of this opportunity. Never has it been so easy to have your donation increased through the United Way, CFC, or your employer’s matching gift program. Learn more and watch your gift grow!
My Voice: Michael Counterman, 20 years old
Michael Counterman is a 20-year-old at the University of Massachusetts in Boston. He has worked with PYD, an inclusion-based organization that helped him interact with other kids and raise awareness about Duchenne. Recently recognized for his ongoing contribution to the group, Michael is an inspiration to anyone who needs a little help finding their niche. He is a natural born teacher, huge sports fan, and an all around fun guy. We’re grateful to have him as our Featured Voice this November. Meet Michael Counterman.
Upcoming Events
• November 10: Grand Rapids .1K
• November 17: Gunnar Listerman Memorial 5K Run, Walk, & Roll for Duchenne
• November 17: Rally for Ryan
• November 21: Hustle for Muscle
• November 30: Swim-a-thon for Duchenne
• December 1: Dart Down Duchenne
• December 6: End Duchenne Gala
• December 7: Hope for the Holidays
• December 15: FACES of Western New York
Check out our full Calendar and sync it with your personal calendar!
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 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.
Facing Reality
"It isn’t easy urging a child to work harder or look further into the future when you’re unsure of what tomorrow will bring, but it’s the only way I know." Parent Advocate & Duchenne Dad, Brian Denger blogs about changes, challenges, and hope in the Duchenne community.
Transforming Duchenne Care: Update
The PPMD has been meeting with the Cystic Fibrosis Foundation and the Muscular Dystrophy Association as part of our follow up to our ongoing care initiative. These meetings have been great next steps to help define our Transforming Duchenne Care mission and to continue work launched at this year’s Annual Connect Conference. Learn more from PPMD’s Director of Care, Kathi Kinnett.
Two Moms, Co-Blogging: What IS Duchenne?
The way we define a word can change our whole perspective. How do you define Duchenne? In this month’s co-blog, Colorado FACES coordinator and parent Ivy Scherbarth and PPMD President Pat Furlong discuss their definition of Duchenne and what that definition means in your day –to-day life. After you read their co-blog, take a minute to share with us how you define Duchenne.
Congratulations & Thank You
Congratulations to this year’s winner of the American Public Health Association Disability Section Lifetime Achievement Award, Dr. Gloria Krahn. Dr. Krahn and her associates at NCBDDD have been extremely supportive of the neuromuscular community. Read more about this friend of our community.
Other Education Updates
Preparing for Emergencies: A Checklist for People with Neuromuscular Diseases
From HerSelf First – The Invisible Constituency: Who Campaigns for Caregivers?
The Flu Vaccine & Duchenne: What You Need to Know
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
With Thanksgiving comes the beginning of holiday shopping, including the two biggest sales of the year: Black Friday (11/23) and Cyber Monday (11/26). 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 ShopForOurSons.org and click the Amazon link. 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
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© Parent Project Muscular Dystrophy 2012 — All Rights Reserved.

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