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Transforming Duchenne Care
April 2014
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I don't think any parent in this community would deny the importance of comprehensive care for people with Duchenne. In fact, we think it's so important at PPMD, that it is part of our mission statement. For the last 20 years, we have tried to identify gaps in care and then put together the right team of people to fill in these gaps.

I am so proud of the launch of PPMD's Certified Duchenne Care Center Program. This new program helps to ensure that centers around the country maintain the highest standards in clinical and sub-specialty services, rapidly apply new evidence based knowledge, and comply with standards in clinical care that were established by the CDC Care Considerations. The goal of the program is to reduce discrepancies in care and to make comprehensive care accessible and available for all patients with Duchenne.

We recently named Nationwide Children's Hospital the first Certified Duchenne Care Center. Over the coming year, plans are being made to award other qualified centers.

We hope you will continue to turn to PPMD with your care concerns by visiting our website. Or, if you are looking for answers that you can't find, contact our Vice President of Clinical Care, Kathi Kinnett, MSN, CNP with your questions.


Pat Furlong
PPMD Founder & President


Research
20th Annual Connect Conference
This year marks PPMD's 20th Annual Connect Conference in Chicago, June 26-29, 2014. The PPMD Annual Connect Conference helps shape our understanding of the Duchenne landscape and brings us closer to the day we end Duchenne.

PPMD's Connect Conference prides itself on bringing together the most important thought leaders in the Duchenne space. But just as importantly, we hope you will find this conference as a chance to really connect with this passionate and supportive community – a community few of us chose to be a part of, but none of us can live without. Register you and your family today. We look forward to seeing you in Chicago!
Webinar: Prosensa Update on Drisapersen
Prosensa presented a status update on drisapersen and its follow-on compounds in a patient focused webinar hosted by United Parent Projects Muscular Dystrophy (UPPMD) on March 25. If you missed the webinar, click here for the presentation.
PPMD Sponsors ENMC Workshop on Biomarkers
In January, PPMD helped sponsor the European Neuro Muscular Centre's (ENMC's) workshop on biomarkers in Duchenne. We were happy to help fund this important meeting that gave the international community an opportunity to look at biomarkers and share experiences and results among colleagues working in Duchenne and other neuromuscular disorders. Read a detailed report from this meeting.
Other Research News
PPMD Awards $100,000 in Exploratory Grants
DuchenneConnect Receives $970,000 Grant
Congratulations to Steve Wilton on winning the 2014 LabGear Australia Discovery Science Award
Exon-Skipping Therapy: A Roadblock, Detour, or Bump in the Road?
Download the latest PTC Therapeutics Webinar hosted by PPMD
Visit PPMD's Duchenne Research News Section for the latest Duchenne Headlines
Advocacy
Success on Funding Letter
Thanks to your efforts, we closed the House and Senate funding letters with the support of 30 House members and 12 Senators. Great work! We appreciate everyone who sent emails and made calls to their members of Congress. These members collectively sent a strong message to the House and Senate appropriations committees who produce the bills that fund the federal agencies that impact Duchenne: NIH, FDA, and CDC. Their message – Duchenne MUST remain a federal priority. See who signed on.
Time to Turn Our Efforts Back to MD-CARE Act
We must now pivot back to securing cosponsors for the MD-CARE Act. To date, we stand at 94 House cosponsors and 25 Senate because of your efforts. As a goal for for April, let's secure 100 House members and 30 Senators! Take action now and find out if your member is on board yet.
PPMD Presents at MDCC Meeting
PPMD President Pat Furlong and Parent Advocate Brian Denger presented last week at the Muscular Dystrophy Coordinating Committee (MDCC) meeting in Bethesda, Maryland. The MDCC is a Congressionally mandated committee designed to coordinate research activities across NIH and with other Federal health programs and activities relating to the various forms of muscular dystrophy (one of the many reasons why the MD-CARE Act is so important). Representatives from CDC, DOD, NIH, and FDA were present along with other stakeholders from the muscular dystrophy community. Pat reported on the development of the FDA guidance and as the current Duchenne representative on the committee, Brian provided an update on PPMD's activities, as well as, reported on other progress in the Duchenne community.
More of Mr. Stalling Goes to Washington
Last month we shared with you a great video capturing the D.C. adventures of Cory Stalling at PPMD's Advocacy Conference. His father, David Stalling, describes the trip in a recent blog, recounting the impact of their Hill visits and how Mary-Lou Weisman's book changed his life. Read the blog.
Other Advocacy News
YES! Thanks to each and every one of you who signed the petition and encouraged others to sign, this community reached well over 100,000 signatures...and more are coming! We continue to prove the power of our collective voice and we look forward to a response from the White House.
In Support of Approvals: PPMD is doing everything it can to enable the FDA to speed the approval of new therapies to all who so desperately need them. Here are 3 simple actions you can take to help.
Community
My Kind of Town...Chicago!
We will have a packed agenda for this year's 20th Annual Connect Conference in Chicago. But during down time, you're going to want to explore this amazing city. (And you'll appreciate the perfect location of the Marriott!) Click here to see a list of things to do provided by the hotel. As we get closer to the Conference dates, we will continue to pass along details of various events and attractions you and your family be interested in. Make sure you are registered today!
Summer Traveling
Before you know it, summer will be here. And now that we are beginning to see real signs of spring, many of us are making early plans for the summer. But what considerations do you need to make when traveling with a child who has Duchenne? How does that determine your choices for summer vacation? What advice would you have for families traveling for the first time with a scooter or wheelchair? We want to hear from you. Join us on PPMD's community site and share your stories and advice with other families who can learn from your family's experiences.
Bike to End Duchenne
Join us for the Million Dollar Bike Ride, a Philadelphia-based bike ride on May 3rd to raise money for Duchenne and other rare diseases. Choose from an 11 mile, 34 mile, or 75 mile course and enjoy the scenic city of Philadelphia in a whole new way! Thank you to The Penn Medicine Center for Orphan Disease Research and Therapy (CODRT) for supporting PPMD in this great endeavor! Read more.
Run For Our Sons
Run For Our Sons Goes International
We're thrilled to announce the addition of the GoodLife Fitness Run Victoria Marathon to our race calendar! Join us in beautiful Victoria, British Columbia on October 12, 2014 for the full marathon, half marathon, 8K, or Kids Run and be a part of our first-ever international marathon team. You'll enjoy discounted hotel rooms within walking distance of the start/finish line, invitations for you and guest to our private pasta dinner, and an official Run For Our Sons tech shirt. Most importantly, you'll raise awareness about Duchenne and give hope to those living with this disease. Register today and go the distance to end Duchenne in British Columbia!
Calling All End Duchenne Super Heroes!
This is the race for you! Join the Avengers team of Captain America, Thor, Hulk, Black Widow, Hawkeye, and Tony Stark for the first-ever Avengers Super Heroes Half Marathon Weekend at the Disneyland® Resort – the extraordinary course on Earth hosted by Earth's Mightiest Heroes! We need your superpowers in the half marathon or Family Fun Run 5K. Every Run For Our Sons team member gets an End Duchenne cape to wear during the race, and we'll gather for a special team pasta dinner during the weekend. Harness your super powers to help us end Duchenne!
Bank of America Chicago Marathon
We have a handful of entries left for the Bank of America Chicago Marathon. Join us on Sunday, October 12th for this iconic marathon through the Windy City. Charity entries are the only way into this race after a competitive lottery drawing, so grab your entry today before they are all gone! We need you to help us make this year's race more successful than ever!
Now recruiting:
Million Dollar Bike Ride - May 3, 2014
Cincinnati Flying Pig Marathon & Half Marathon - May 4, 2014
DICK's Sporting Goods Pittsburgh Marathon - May 4, 2014
Kaiser Permanente Colfax Marathon - May 18, 2014
Rite-Aid Cleveland Marathon & Half Marathon - May 19, 2014
Rock 'n' Roll Seattle Marathon & Half Marathon - June 21, 2014
San Francisco Marathon - June 27, 2014
Disneyland Half Marathon Weekend - August 29 – 31, 2014
Bank of America Chicago Marathon - October 12, 2014
GoodLife Fitness Run Victoria Marathon - October 12, 2014
Baltimore Running Festival - October 18, 2014
TCS New York City Marathon - November 2, 2014
Avengers Super Heroes Half Marathon Weekend - November 14 – 15, 2014
Walt Disney World Marathon Weekend - January 8 – 11, 2015
And Save the Date for these popular 2015 races!
• Chevron Houston Marathon & Aramco Houston Half Marathon - January 18, 2015
• Rock 'n' Roll New Orleans Marathon & Half Marathon - January 25, 2015
Simple Actions Have Lasting Impact
When you believe in a cause, when you find an organization that feels like home, you want to show your support but you don't always have the time. Especially when you are navigating the day-to-day care of someone in your life with Duchenne. So sometimes the simplest action you can take that will have the greatest impact is a donation.

PPMD's STIR program is a monthly giving opportunity, 100% of which goes towards funding research. This is a chance for not just you, but your entire family, to support potential therapies at a dollar amount you are comfortable with. In fact, we have a few PPMD families where every single relative (grandparents, aunts, uncles, cousins, and siblings) are giving. These are families that may not be able to pledge a large gift to PPMD. But by joining STIR and encouraging others to do the same, their combined monthly gifts are having an incredible impact.

Consider STIR and know exactly where your gift goes.
Upcoming Events
• May 3, 2014: Imagine How Music Festival
• May 3, 2014: The Million Dollar Bike Ride
• May 4, 2014: Flying Pig Marathon
• May 4, 2014: DICK'S Sporting Goods Pittsburgh Marathon
• May 17, 2014: Kids Dash for Micah's Miracle
• May 17, 2014: Team Aiden 5K
• May 17-18, 2014: Kaiser Permanente Colfax Marathon
• May 18, 2014: Rite-Aid Cleveland Marathon
• May 18, 2014: The Millennium 5K 4 Jake's Sake
• May 27-30, 2014: Spike Duchenne
• June 21, 2014: Rock 'n' Roll Seattle Marathon & Half Marathon
• June 26-29, 2014: 2014 Annual Connect Conference
Check out our full Calendar and sync it with your personal calendar!
Connect Conference: Register Now!
2014 Connect Conference in Chicago
Date: June 26-29, 2014
Location: Chicago Marriott Downtown Magnificent Mile
Registration is Open!
Education
NEW Certified Duchenne Care Center Program
PPMD is proud to award Nationwide Children's Hospital as our first Certified Duchenne Care Center. Every person with Duchenne deserves the best care and treatment possible, and has the right to know what clinics meet optimal standards. This program will do just that. Find out more about the program's goals, strategy, and next steps.
A New Care Guide to Give to Your Provider –
Imperatives for Duchenne Care
Health care providers care for many people, with many diagnoses – far too many for any one provider to be an expert in them all. Make sure that you are receiving the best possible care by giving your provider this NEW "cheat sheet" guide to comprehensive Duchenne care.
Education, Behavior, and Duchenne
PPMD FACES Coordinator and Education & Behavior Specialist, Jill Castle, has launched a blog for families to provide behavioral and education support. Jill is the mother of two boys – Oliver and Anthony (who has Duchenne). This new blog will help you navigate your child's school, interact with teachers and administrators, and understand some of the emotions your child is going through. Check out the blog and feel free to reach out to Jill with questions and comments.
Living Duchenne: Ongoing Traumatic Stress
In her monthly blog, Living Duchenne, Colorado/Wyoming FACES Coordinator Ivy Scherbarth looks at the continuing stress of a Duchenne diagnosis in the life of a family, and the need to choose joy over trauma. After you read Ivy's Blog, tell us how you find the joy in a stressful and emotionally draining day.
Navigating Insurance Issues Webinar
Health insurance coverage for patients with rare diseases has undergone significant changes in recent years. At the forefront are changes in how the insurance industry designs and delivers benefits and new coverage options created by the Affordable Care Act. Find out how you can better evaluate what options are best suited for your individual needs by joining the Global Genes Project on April 30th.
More Education Updates
Results from To Tell or Not to Tell – That is the Question: Read the latest blog from Mary-Lou Weisman and find out how other parents responded.
Your Clinical Trial Experience: We're working with industry to change the way clinical trials are designed and conducted in the Duchenne community, and we need YOUR input in order to report back.
Antimicrobial from soaps promotes bacteria buildup in human noses
Simple Actions
Registration is now open for PPMD's 20th Annual Connect Conference in Chicago, June 26-29, 2014. Join us for the single most important meeting in the Duchenne community, as top researchers, physicians, industry, and care providers come together to share the latest news on promising therapies, clinical trials, and care. This is your direct connection to top thought leaders in Duchenne, as well as families just like yours who are navigating the world of this disease.
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 2014 — All Rights Reserved.

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