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Simple Actions, Lasting Impact
December 2013
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There are days when the stars align just right. Seems to me, December 12, 2013 was one of those days.

I just wrote a blog about last week's Duchenne Policy Forum. This was a historical moment in our community and a meeting that holds a great deal of promise for our community. It was rather simple. We talked and the FDA listened...really listened. They asked questions, we asked questions, and everyone shared answers, possibilities. We all left with marching orders and next steps. It's what a meeting – a good meeting – should be. Dialogue, team work, community.

It took weeks/months of planning, lots of logistics, lots of back and forth. But in the end, what we accomplished was actually rather simple: Next steps with an important partner.

We are deep in the midst of our holiday campaign raising money for the Duchenne Superhighway. This is another idea that, at its core, is relatively simple. But we need the continued support of everyone reading this to help get this project off the ground and hopefully, in turn, speed up the clinical trial process.

We do this and you do this for the heroes of this community – those that we hold so dear in our lives who have Duchenne. They are, quite simply, our hearts. In this month's newsletter our Featured Voice is your son. And your daughter. And your nephew. And your granddaughter. Anyone with Duchenne.

Simplicity. It's a word that seems almost foreign, especially this time of year with the hustle and bustle of every day life compounded by the holidays. We appreciate you and your willingness to find that treasured spare moment and take a simple action to help end Duchenne.

It's been a fascinating year – sometimes great, sometimes tragic, but never dull. We feel a renewed energy and fight heading into 2014 after last week's Policy Forum though. And we look forward to working with you to make it a redefining year for the Duchenne community.

Warmest holiday wishes from my family to yours,


Pat Furlong
PPMD Founder & President


Research
PPMD to Launch Duchenne Superhighway
There is no lack of ideas or interest – 7 public companies and more than 15 biotechs are focused on finding treatments for Duchenne. We need to actually change the landscape and bring drugs to market faster. It's from this need that the idea for the Duchenne Superhighway was born.

The purpose of the Duchenne Superhighway is a simple one: identify and eliminate bottlenecks in the clinical trial process that slow things down and don't add value. With such a full pipeline, we need to make sure that potential therapies can be tested as efficiently and quickly as possible. The Duchenne Superhighway will help to make this testing process faster and simpler. Learn more about the Duchenne Superhighway.
Attend the Duchenne Superhighway Webinar
To help you understand the necessity and goals of the new Duchenne Superhighway project, PPMD will be hosting a webinar with guests from Halo Therapeutics, PTC Therapeutics, and Sarepta Therapeutics on Thursday, December 19 at 1pm eastern. View the webinar details.
Getting There Faster
Late last month we sat down with large and small companies in the Duchenne space to better understand their needs to conduct clinical trials for drug development. This meeting follows on the heels of a $300,000 commitment by PPMD to the Duchenne Superhighway project (in collaboration with DART Therapeutics) to strengthen the clinical trial infrastructure in the Duchenne space so that trials can be conducted faster and more efficiently. Read a meeting update by PPMD's Vice President of Research, Sharon Hesterlee, PhD.
Decode Duchenne Officially Launches
PPMD and DuchenneConnect, a program of Parent Project Muscular Dystrophy, announce a genetic testing program for patients with Duchenne or Becker muscular dystrophy. The program, called Decode Duchenne, is supported by Sarepta Therapeutics. It provides genetic testing at no cost to eligible patients who are unable to access testing due to barriers such as a lack of insurance or insufficient insurance coverage. Find out how you can participate. Thank you to Sarepta for supporting this important new program!
This Month in Muscular Dystrophy
Hosted by Kevin Flanigan, MD, of Nationwide Children's, "This Month in Muscular Dystrophy" podcasts highlight the latest in muscular dystrophy and other inherited neuromuscular disease research. This month, Louis Chicoine, MD, principal investigator in the Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital; assistant professor of Pediatrics at The Ohio State University College of Medicine discusses the effect of Plasmapheresis in the removal of AAV antibodies for gene therapy. Download the podcast now.
Other Research News
Webinar Recording: Update on Natural History Data Study from November 19 with Craig McDonald, MD
New muscular dystrophy treatment shows promise in early study led by Children's National
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
Visit PPMD's Duchenne Research News Section for the latest Duchenne Headlines
Advocacy
PPMD Hosts Landmark Duchenne Policy Forum
PPMD was proud to host an historical Duchenne Policy Forum on December 12, 2013 in Silver Springs, MD. Entitled "Optimizing Clinical Trials in Duchenne Muscular Dystrophy in the New Era of Improved Care Standards: Guidance for the FDA", the meeting was designed to create a draft guidance for the FDA that reflects the combined experience and wisdom of the Duchenne community. We couldn't be more happy with the discussion that was started with representatives from FDA and we are grateful to everyone in the community who participated.

For those of you unable to attend and want a recap, please read an in-depth report from PPMD's Vice President of Research, Sharon Hesterlee, PhD.

It's not too late for you to share your thoughts, questions, and experiences with the FDA. Share Your Story and read stories already submitted.
We STILL Need Your Help with the MD-CARE Act
PPMD is still actively gathering cosponsors for the MD-CARE Act. Ryan Fischer, PPMD's Director of Advocacy and Outreach updated his blog reflecting 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.Take a minute during the holiday season, reach back out to your representatives on Capitol Hill, wish them a Happy 2014, 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. The hustle and bustle of the holidays can't slow down our momentum for this critical legislation. This is one of simple action anyone can take! Read Ryan's blog to learn more.
PPMD President Interviewed by Rare Disease Report
Before last week's Duchenne policy forum, Pat Furlong sat down with the Rare Disease Report to talk about the need to optimize clinical trials in Duchenne. The interview with Pat was conducted at the First Annual Fall Retreat hosted by the Center for Orphan Disease Research & Therapy (CODRT) at Penn Medicine. Watch her interview here.
Save the Date!
PPMD's Annual Advocacy Conference
Date: February 23-25, 2014
Location: Marriott Wardman Park
Save the Date
Other Advocacy News
PPMD Board member Lance Hester explains why last week's FDA Policy Forum is important to every Duchenne family - from rare mutations to Exon 51
Help Update the MD Action Plan - Please check out Brian's recent blog and read about how you can help inform the update
Community
Our Olympic Champ with the Gold Heart
PPMD supporter and Olympic champion Ryan Lochte continues to use his international fame for the Duchenne community! His latest endeavor with Speedo USA's Art of the Cap campaign kicked off last week and we couldn't be more thrilled to be one of five charities featured! Art of the Cap features five Olympic swimmers and five inspiring artists collaborating on limited-edition swim caps for worthy causes (including PPMD). Get yours on SpeedoUSA.com before they're gone! These are one-of-a kind items designed by Ryan with a portion of all proceeds going to PPMD.

And if that wasn't enough, Ryan has partnered with Yogurtology to launch two exclusive new frozen yogurt flavors with sales benefitting PPMD. Visit your local Yogurtology and support the fight to end Duchenne by trying out these exclusive new flavors today!

Thank you Ryan for all that you do to help end Duchenne!
One Simple Action Can Score a Touchdown!
Division III Coach Mike Rader from Maryville College in Tennessee is up for Liberty Mutual Coach of the Year. The winner of this contest gets $50,000 for the charity of his choice – Coach Rader has chosen PPMD! Please take a minute to vote through Facebook and help Coach To Cure MD and Coach Rader
Help PPMD Win!
Vote for Parent Project Muscular Dystrophy as your favorite charity and help us win $50,000! Cast your vote and then SHARE with your friends to help spread the word. The charity with the most votes wins. Visit Run for a Reason to cast your vote today!
Run For Our Sons
Be a Duchenne Community Superhero!
Our runners amaze us every day with their can-do attitudes, perseverance, and commitment to helping us end Duchenne. So in 2014, the 10th year of the Run For Our Sons program, we are recognizing all of our superheroes by providing them with capes! These caps identify our runners as superheroes in our community, using their special powers to run for those who can't. By running to end Duchenne, our runners are helping to make the impossible possible, as true heroes do! Sign up for a 2014 race today and get your End Duchenne superhero cape!
Run With Us in New Orleans
New Orleans is like no other city in the world, and you can experience it in a whole new way by running the Rock 'n' Roll New Orleans Marathon! Join us February 2nd for the full marathon, half marathon, or half marathon relay on a flat course through the city's historic neighborhoods and the famed French Quarter. This is one of our fastest growing teams and we hope you'll join us!
Register today.
Runner in the Spotlight
Running a marathon is no easy feat, but running two marathons on back-to-back weekends? Some might call that a heroic undertaking! This month's runner in the spotlight, Mark Brill, ran the Marine Corps Marathon and the ING New York City Marathon on consecutive weekends this fall, in honor of his wife's two cousins and all those living with Duchenne. Meet Mark.
We need YOU!
Now recruiting for:
Rock 'n' Roll New Orleans Marathon & Half Marathon - February 2, 2014
Cincinnati Flying Pig Marathon & Half Marathon - May 4, 2014
Rite-Aid Cleveland Marathon & Half Marathon - May 18, 2014
Rock 'n' Roll Seattle Marathon & Half Marathon - June 21, 2014
Our Voices
Each month for the last couple of years we have featured people with Duchenne who have made an impact on their community and our community in some significant way. Whether working with their parents to host a fundraising event, participating in a race, speaking in Washington, D.C., or sharing their story creatively to raise awareness, all of the amazing people we have featured have had an impact.
(Meet some of our past Featured Voices)

This month we would like to turn to you and tell us who you would like to tell the community about. Who is the incredible person in your life who has Duchenne and what have they done to educate and change their community? Let's end the year celebrating these special people in our lives!
Upcoming Events
• January 3, 2014: 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: 2014 Rock 'n' Roll New Orleans Marathon & Half Marathon
• February 15, 2014: UF Student Physical Therapy Association Golf Outing
• February 23, 2014: 2014 Annual Advocacy Conference
• 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
Education
Cardiac Care: Steps to Incorporate in Your Child's Care
Even if you are doing everything right in terms of care, cardiac issues in people with Duchenne and Becker are not just possible, but likely. And while we can't prevent all scenarios, we want to remind you of a few basic steps you should be incorporating in your child's care. Learn more.
Siblings: Life with Duchenne
Intensive Care: A Family Love Story author Mary-Lou Weisman recently asked to hear from siblings of people with Duchenne, wanting to better understand how they were affected by the experience of living with & loving a sibling with the condition. Were they more compassionate? Angry? Scared? Guilty? Read what she learned and join the discussion on Facebook about your experience as a sibling or what you see happening in your own family.
Living Duchenne: In Loving Gratitude
"I like holidays and parties and lights and libations. I like travel. I like family. But some midwinters just don't have that feeling for me. I think instead that I would like to spend the year's longest nights all alone in silent contemplation beneath big brassy wintertime stars. Alone. With nobody bothering me. Bah! and Humbug!" Read more of Ivy Scherbarth's latest guest post, "In Loving Gratitude." Check out Ivy's blog.
More Education Updates
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.
Make it Possible!
Simple Actions Go Far
We need you to take one simple action today: Donate to help us raise $200,000 for a project that will speed up drug development.

And because our Board of Directors has pledged to match your donation dollar for dollar, your gift is one simple action with twice the impact!

Take one simple action!
Shop For Our Sons
There's still time to order through Amazon, but the clock is ticking! 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.

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.
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 2013 — All Rights Reserved.

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