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It's Time to Connect
June 2013
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Next week, hundreds of us will descend on the beautiful Baltimore waterfront for PPMD's Annual Connect Conference.

For many of us in the community, June means that it is time to reunite with friends (and Duchenne experts that have become friends!). For some, this may be your first time experiencing a sometimes overwhelming but always informational event like no other in our community. And for others, we know that circumstances might not allow you to attend this year's Conference, but we hope you'll connect virtually by watching presentations online and following us on Facebook and Twitter.

At this year's Conference, amidst the researchers, scientists, and industry presenting, will be 20-year-old Ben Dupree who lives with Duchenne and is our Featured Voice for June. Ben is himself a promising researcher, focusing his studies on computer assisted drug discovery and design. I hope you'll take a minute to meet this extraordinary young man.

I am always particularly proud of the work PPMD is doing when the Connect Conference rolls around. It gives us an opportunity to look back at the progress made since the last time the community gathered. And it helps us focus on the work that needs to be done moving forward. To help illustrate what we have accomplished, I am pleased to present PPMD's Progress Report. This is a comprehensive look at all of our various programs and a chance for you to see and understand how each thing we do as a community affects our ultimate outcome – to end Duchenne. I hope you'll take a moment to look at what we've done and how you've helped to make this progress possible.

I look forward to connecting and reconnecting next week!


Pat Furlong
PPMD Founder & President

Research
Updated Clinical Trial FAQ Sheets
Are you confused by all the research going on right now? Are you having trouble keeping all of the clinical trials straight? If so, you are not alone! DuchenneConnect, a program of PPMD, has created a summary of Frequently Asked Question (FAQ) sheets about all clinical trials involving Duchenne and Becker, as well as research that is soon to be in clinical trial. The FAQ sheets will be part of the program book at PPMD's Annual Connect Conference and they are now housed on the DuchenneConnect website for your convenience. Click here to view them.
Now Recruiting
Ataluren Phase 3 Trial Now Recruiting
The Phase 3 clinical trial for ataluren is now recruiting at one site in the US: Cincinnati Children's Hospital Medical Center in Cincinnati, Ohio. There will be over 50 trial sites in at least 18 different countries. Ataluren is an investigational new drug developed by PTC Therapeutics for nonsense mutation Duchenne muscular dystrophy. To read the full details about this trial, including enrollment criteria, please click here to read a FAQ provided by PTC Therapeutics.
FOR-DMD Trial Now Recruiting in the US, Canada, and the UK
FOR-DMD is now recruiting in the United States, Canada, and the United Kingdom. Additional sites will be opening soon in Italy and Germany. The purpose of this study is to determine the best regimen of prescribing corticosteroids to boys with Duchenne. FOR-DMD will compare three ways of prescribing steroids to determine which of the three ways increases muscle strength the most, and which causes the fewest side effects. Learn more about the study.
Duchenne Research News in One Place
PPMD has created a research hub that you should bookmark as your go-to resource for research updates. On PPMD's homepage under Duchenne Research News and powered by Scoop.it!, this is like Google Alerts for Duchenne news! We will continue to provide context to these updates when necessary so that you know how this news affects the community more specifically. So please take a moment to check out PPMD's new Duchenne Research News section today!
Other Research News
PPMD Launches Duchenne Central App
Wall Street Journal: New Muscular Dystrophy Drugs Offer Hope
Visit PPMD's Duchenne Research News Section for the latest Duchenne headlines
Advocacy
Benefit/Risk Survey Results to Be Submitted to FDA
Last week, PPMD hosted a webinar to discuss our FDA "aggressive collaboration" approach and the results from our recent benefit/risk survey. We conducted a rigorous survey of more than 100 parents whose children have Duchenne, gathering invaluable perspectives as to treatment priorities and risk threshold based on realistic but hypothetical treatments – information that PPMD is encouraging regulators to use to accelerate review time and, ultimately, to speed access to effective therapies. If you missed last week's webinar, click here to learn more about the results and how you can still share your story with the FDA.
Continuing the Quest for Accelerated Approval
PPMD President Pat Furlong continues her blog series defining the importance of each recommendation outlined in Putting Patients First, a white paper outlining recommendations to speed responsible access to new therapies for Duchenne.

And if you missed last month's webinar discussing PPMD's white paper, click here to watch the presentation.
ACTION ALERT: It's Time to Reach Back Out!
Hundreds of you have already met with, phoned, or emailed your Members of Congress to ask them to cosponsor the MD-CARE Act Amendments of 2013. But some still have yet to sign on. See if your Member has signed on. Let's remind them how important this legislation is to our families! Each signature brings us closer to continuing the important research for muscular dystrophy.
More Advocay Updates
PPMD is proud to be featured in an article from Inside Health Policy about the need for accelerated approval.
Rare Disease Report Covers PPMD's New White Paper
Community
Run For Our Sons
WOW!
Thank you to the 2013 Run For Our Sons team, from our 5Kers to our Marathoners. In just six months, we have had over 1,600 runners participate in races this year, raising over $545,000! We can't thank you enough for going the distance to end Duchenne – and there's still half a year left!
First Hand Account of the Boston Marathon
"Patriots' Day, Monday, April 15, 2013 is a day we won't forget. Much has been eloquently written about the day after 2:49pm." –Chris Hancock, RFOS Runner
Read his experiences prior to the tragedy.
Race Recap –
Rite-Aid Cleveland Marathon
For the second year in a row our Cleveland-area runners and walkers of all ages competed in the full & half marathon, 10K, and 5K races to raise much needed funds and awareness and help to end Duchenne.
Thank you to each and every runner who made this year's race so successful! Check out some team photos from this weekend.
We need YOU!
Now recruiting for:
Rock 'n' Roll Seattle Marathon & Half Marathon - June 22
Disneyland Half Marathon, 10K & Family Fun Run 5K - August 31 & September 1
Medtronic Twin Cities Marathon - October 6
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
ING New York City Marathon - November 3
Naperville Marathon - November 10
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
Post Conference Fun!
Looking for things to do in Baltimore after sessions? Here are some links worth checking out:

• The NY Yankees will play the Baltimore Orioles, June 28, 29 & 30! Buy tickets now if you are interested in attending Friday or Sunday (Saturday is the PPMD Dinner!). Note, students can get $6 tickets in the upper level for Friday, 6/28, subject to availability. Buy tickets now.

• Receive discounts and coupons to some of Baltimore's best restaurants, shopping, and attractions. Learn more.
My Voice: Ben Dupree, 20 years old
Coach To Cure, Sam's Day, North Texas FACES group, and now participating at PPMD’s Connect Conference at the end of June in Baltimore. These are just a few of the things 20-year-old Ben Dupree and his family participate in on behalf of the Duchenne community to raise awareness and money for Duchenne research. As an adult, though, Ben is also participating in PPMD's pre-conference meeting concerning transition care. This generation of people with Duchenne are redefining what it means to be an adult living with Duchenne and Ben is adding his unique perspective. Meet our Featured Voice for June, Ben Dupree!
Upcoming Events
• June 17: Jumping Jack Golf Tournament
• June 20: Tee Off For Tanner
• June 22: Rock 'n' Roll Seattle Marathon & Half Marathon
• June 22: Jared Sweet Golf Tournament
• June 27-30: Annual Connect Conference
• June 29: Team Aiden's Family Fun 5K
• June 29: Dance for Duchenne
• June 27: Friar Fight to End Duchenne Golf Tournament
• September 28: Coach To Cure MD
Check out our full Calendar and sync it with your personal calendar!
More Community Updates
• In 2013, the Northwestern University Dance Marathon raised over $1.2 million for their charitable partner. And in 2014, they will be raising funds for Duchenne! Thanks Team Joseph for all of the hard work you do for this community!
Education
PPMD Launches Care for Duchenne Section
Based on input from parents and people with Duchenne, PPMD has created a new and improved Care for Duchenne section on our website. Learn more about the revamped site, including urgent care updates and the reorganization of information by areas of care and by stages of Duchenne.
Dental Dilemmas in Duchenne
When a person with Duchenne has general anesthesia, serious problems may arise. PPMD's Vice President of Clinical Care, Kathi Kinnett reached out to experts to find out what you need to know in order to keep you (or your child) safe when treating dental issues using anesthesia. Read Kathi's blog.
Living Duchenne: Expertise
You are the expert. FACES Coordinator Ivy Scherbarth explains why it is so important that you trust your own judgment, reason, and authority when it comes to your child's care. Read Ivy's latest blog.
Code of Ethics in Patient Education
Sarah Krug of CANCER101 shares an interesting new essay about the need for structure in patient education.

"The "participatory medicine" movement is underway where networked patients are shifting from being mere passengers to responsible drivers of their health, and clinicians are encouraging them as full partners… Patients are key decision makers and should be given the equivalent of what healthcare professionals receive, but in laymen's terms."

Read the essay.
More Education Updates
DuchenneConnect Presents 2012 Data & 2013 Update
Download PPMD's updated emergency care card featuring revised information on giving oxygen to people with Duchenne.
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
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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