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Parent Project Muscular Dystrophy Donate Now
Connecting You to Clinical Trials
March 2013
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Spring is in the air. That’s not to say there isn’t still snow falling in some places, bitter temperatures, and gray skies. But every now and then,
hopefully like me, you are starting to see hints of warmer weather and greener pastures.

Spring is about rebirth and cleaning house…at least in my mind. And I think it’s the perfect time to introduce you (or perhaps reintroduce you) to DuchenneConnect, a program of PPMD that we are quite proud of. Did you know that DuchenneConnect is one of the most important tools in your arsenal of resources in the fight to end Duchenne? The most comprehensive, Duchenne-specific registry of its kind, DuchenneConnect links your son or daughter to upcoming clinical trials and studies that may lead to the development of new therapies and improve our understanding of Duchenne.

Now is the time to register with DuchenneConnect. And if you are already registered, make sure your child’s information is up to date. Even if you don’t want your child to participate in a clinical trial necessarily, the information you share will be used by researchers to improve care, advance research, and speed treatments for Duchenne. Your participation in DuchenneConnect can make a difference and is one of the ways you can help end Duchenne.

Christopher Cameron and his family understand the value of DuchenneConnect. Christopher is our Featured Voice for March and has been participating in natural history study that is proving to be an invaluable project for Duchenne research. Meet this amazing, selfless young man.

Sunny skies are coming for all of us – literally with the approach of Spring, but also figuratively as we continue to make huge strides in our fight to end Duchenne.

Warm wishes,


Pat Furlong
PPMD Founder & President

Research
PPMD Funds Project to Identify Genetic Changes that Affect the Severity of Duchenne
PPMD will provide $100,000 to Dr. Stanley Nelson of the University of California, Los Angeles, to examine the genomes of 40 boys with Duchenne who are either very mildly affected or very severely affected. The goal is to identify changes in genes other than dystrophin that may affect the course of the disease. Read the release.
Stem Cells Regenerating Muscles in Duchenne Mice
Investigators are moving closer to the time when cells from a person with Duchenne can be removed and converted into ever-renewable source of healthy cells to repair the damage caused by the disease. This article explains the work of Rita Perlingeiro at the University of Minnesota, who was recently awarded $220,000 from PPMD to pursue this strategy. Learn more about the study.
PTC receives funds to support Phase 3 confirmatory
trials of ataluren
PTC Therapeutics, Inc. (PTC) announced the successful completion of a $60 million financing led by Brookside Capital Partners Fund, L.P. Proceeds from this financing will support continued clinical development of ataluren in Duchenne. PTC is initiating a Phase 3 confirmatory trial of ataluren in people with nonsense mutation Duchenne with the goal of commencing enrollment in the first half of 2013. Learn more.
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, Dr. Louise Rodino-Klapac discusses Alpha 7 Integrin as a therapeutic approach to muscular dystrophy. Click here for the latest podcast.
Other Research News
Pipeline Update: PPMD received an email from Dr. Rohit Batta following the recent story regarding his presentation on drisapersen at the Duchenne Parent Project Onlus meeting on February 24.
Pipeline Update: Eteplirsen - Sarepta's Q4 & FY12 results, latest developments, and next steps.
Congratulations to our friends at Patient Crossroads on the launch of the CONNECT Open-Access Patient Registry Program. Programs like this will help collect invaluable data on rare diseases.
Advocacy
Update on PPMD’s Risk/Benefit
in Duchenne Therapies Program
We were thrilled that 119 parents/guardians were willing and able to complete the entire treatment preferences survey. We are analyzing the data. We are doing the first part of the data analysis in-house, and the second part with our health economist collaborator. Our plan is to present this information to the FDA, to industry and clinical investigators, and back to you. We’ll also publish this data in a medical journal. BUT, we still need you to share your story to help us make our case to the FDA that much stronger. Learn how you can participate.
A Community United
Parent Project Muscular Dystrophy, The Muscular Dystrophy Association, and the Foundation to Eradicate Duchenne worked closely with their champions on the hill for months in order to introduce the strongest possible piece of legislation. We would like to highlight all the muscular dystrophy groups who agreed to sign onto our multi-organization letter, hand delivered to every member of Congress during our Advocacy conference. The letter sent a strong message to Congress about a united effort in favor of the renewed legislation. Click here to find out more about this letter.
ACTION ALERT: Update on
Reauthorization Efforts & What You Can Do
We continue to make progress on the Reauthorization of the MD-CARE Act. Thanks to you, we have already collected a number of cosponsors for the legislation. It’s a strong start and we must continue to tell our story to congress and explain why renewing this critical legislation is paramount to all those living with Duchenne and all those diagnosed tomorrow and beyond. Please continue to send the message to friends and family and ask them to take action. Read more updates about the Reauthorization of the MD-CARE Act, including which Members have signed on.
Community
Run For Our Sons
Going the Distance to Make it Possible!
January was a phenomenal month for Run For Our Sons and the PPMD community. In one extraordinary weekend, 270 runners participated in two major marathons and raised more than $350,000 to end Duchenne. Our runners, walkers, and rollers in the Walt Disney World Marathon Weekend and the Chevron Houston Marathon kicked off what is sure to be one of the best years yet for Run For Our Sons.

In 2012, Run For Our Sons raised more than $1.3 million – a record-breaking year for our endurance program! Read more about the incredible growth of Run For Our Sons and how your participation makes a difference.
Disneyland Half Marathon, 10K & 5K
Join us in the Happiest Place on Earth for the happiest race on Earth! Labor Day weekend (August 31st & September 1st) we’ll be participating in the Disneyland Half Marathon, 5K and the brand new 10K race! This is one of our most exciting family weekends of the year, beginning with a team pasta party featuring Disney characters and continuing throughout the weekend with races, a finish line tent, hotel discounts, and of course, the Disneyland theme parks for everyone to enjoy. Space is limited so sign up today and let the memories begin!
Marine Corps Marathon
Run For Our Sons is thrilled to be participating for the first time in the Marine Corps Marathon, a historic race through our nation’s capital! Entries into this exclusive race are hard to find; by joining the Run For Our Sons team you’ll receive guaranteed entry and a number of other benefits. Most importantly, you’ll be giving hope to those living with Duchenne. Join us on October 27th. We have a limited number of entries available, so register soon!
Race Recap
Check out photos from the Rock ‘n’ Roll New Orleans Marathon, which took place February 24th. Thirty runners, walkers, and spirit runners came out and raised more than $40,000 for our second year participating in this race. Thank you to everyone who ran, cheered, and sponsored our amazing team of runners!
We need YOU!
Now recruiting for:
Cincinnati Flying Pig Marathon - May 5
Rite-Aid Cleveland Marathon - May 19
Rock 'n' Roll Seattle Marathon & Half Marathon - June 22
Disneyland Half Marathon, 10K & Family Fun Run 5K - August 31 & September 1
Under Armour Baltimore Running Festival - October 12
Bank of America Chicago Marathon - October 13
Rock ‘n’ Roll Denver Marathon & Half Marathon - October 20
Marine Corps Marathon - October 27
My Voice: Christopher Cameron, 11 years old
As we remind you to register for DuchenneConnect or update your information in DuchenneConnect if you are already registered, we wanted to introduce to a young man who has helped solidify the importance of this registry. Christopher Cameron, an 11-year-old from Southern California, is participating in the natural history study and has participated in clinical trials as well. He, with the support of his family, knows the importance of the work being done in Duchenne research and the critical role he can play in furthering this research. It is because of his bravery and selflessness to participate that we celebrate Christopher Cameron, our Featured Voice this March.
Upcoming Events
• March 15: End Duchenne Dance-a-Thon
• March 17: Shamrock Shuffle 5K Run-Walk
• March 24: Afternoon of Music and Fun
• March 30: Painting for Fun
• April 1: Art For a Cure For Duchenne
• April 5: Dancing with the Spartans
• April 7: Pins for Duchenne
• April 20: Unionville 5K
• April 27: Memphis Brewfest
• April 26-28: 2013 West Coast Connect Meeting
Check out our full Calendar and sync it with your personal calendar!
More Community Updates
In the race to end Duchenne, the one thing we don’t have is time. That’s why we need you to be as active as you can in our community. What will you do?
Education
PPMD’s West Coast Connect Meeting
We are just over a month away from PPMD’s West Coast Connect Meeting in San Diego and we are happy to unveil our in-depth agenda for this year’s meeting. Topics will include:

  • Natural history data
  • Talks regarding biomarkers
  • Numerous Duchenne research presentations
  • Care discussions to include topics such as cardiology, bone health and transition care
  • Hands–on PT sessions
  • Discussions related to behaviors and accommodations in Duchenne
  • A panel presentation from some of our industry partners to discuss and update us on clinical trials
  • And we are excited to be putting in place an Expert Panel of young men living with Duchenne who will be presenting on all topics Duchenne.

    Make sure you take advantage of this informative and important meeting. Register today!
Living Duchenne: Let’s Talk About Stress, Baby
Each month, Colorado FACES coordinator and parent Ivy Scherbarth brings you her thoughts related to the Duchenne journey. This month, Ivy talks about the numerous stresses in her life and how she tries to take a moment to breath. Sometimes the greatest comfort is right here in the Duchenne community amongst people on a similar journey. Tell us what the stresses are in your life and how you find ways to manage. Read Ivy’s latest blog.
PPMD Cardiac Webinar Series: Caring for Adults Living
With Duchenne
On March 6, PPMD hosted the final installment of its cardiac webinars. This webinar discussed adult cardiac surveillance, multiple organ surveillance in adults, medical heart failure management, mechanical heart failure management in adults, and questions concerning cardiac transplants. To view this webinar, as well as the other webinars in our cardiac series, click here.

PPMD President Pat Furlong shared her dreams about cardiac solutions that would help all people. Read her blog.
Orphan Drugs Research & Commercialization Conference
PPMD is proud to be a sponsor of the inaugural Orphan Drugs Research & Commercialization Conference in Boston, May 8-10, 2013. GTC’s Conference will bring together all stakeholders in the rare diseases space, from leaders in industry, academia, government, and patient groups, to collaborate and establish better partnerships that will advance scientific development and accelerate clinical research, to improve the lives of those affected by rare diseases. Register today and receive 20% off your registration using the discount code: PPMD2013.
Other Education Updates
Download PPMD’s updated emergency care card featuring revised information on giving oxygen to people with Duchenne.
In the fifth and final article by Siren Interactive to coincide with their rare disease focus month, Siren’s founder and president interviewed Pat Furlong about rare disease research for Rare Disease Day.
Save the Date: PPMD’s Connect Conference, June 27-30, 2013 in Baltimore
Make it Possible!
Connect to Clinical Trials
Do your part – register on DuchenneConnect today! You will learn about actively recruiting clinical trials and research studies and how to participate, and you will help to advance research and speed treatments for Duchenne. Already registered? Please remember to update your Profile Survey at least once a year. Researchers need up-to-date data for their studies and we need this data to know what trials and studies are right for you or your child. Update your Profile now! Questions? Email coordinator@duchenneconnect.org.
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 2013 — All Rights Reserved.

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