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What Do YOU Think?
March 2014
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When my sons were diagnosed, honestly few people cared about my family's experience navigating the Duchenne diagnosis that had shaken our home to the core. As parents, we barely had a voice and even if we screamed, no one was interested in listening. And people with Duchenne? Forget about it. Rarely did a clinician or researcher approach my son or anyone else's child and ask them how they felt about living with Duchenne.

Fortunately, though, it is a new era in Duchenne. Not only do we have a voice, and a loud voice for that matter – but experts are eager to hear what we have to say. They want our children to speak and they are placing value on their firsthand experience with Duchenne.

So it becomes our obligation to take advantage of the scientific community's interest! PPMD, and especially DuchenneConnect, turn to you regularly to fill out surveys or take part in interviews. Some are PPMD studies and many are from other scientists. No, these aren't the 5 minute surveys you take online to find out which Star Wars character fits your personality. These take a little more time, but the information you provide is priceless.


Pat Furlong
PPMD Founder & President


Research
PPMD Awards $100,000 in Exploratory Grants
PPMD is thrilled to award two exploratory grants as part of our ongoing grant program. Peter Arthur of the University of Western Australia was awarded $50,000 to finish testing a molecule called procysteine in animal models in preparation for a human clinical trial. Procysteine is an antioxidant that has been tested in other disorders and has been shown to correct some of the defects caused by the lack of dystrophin in mice.

PPMD has also awarded ReveraGen BioPharma an Exploratory Award of $50,000 to determine if small snippets of RNA called "microRNAs" can affect the efficiency of exon-skipping. The investigators will inhibit the microRNAs that seem to be problematic while introducing antisense oligos to stimulate exon skipping. Learn more about PPMD's grant program.
PPMD Announces Request for Applications to Xcelerate Duchenne Drug Development
Continuing our tradition of pioneering vital Duchenne research, PPMD is proud to announce our latest Duchenne research request for proposals. In recognition of the large number of late stage preclinical and early stage clinical therapeutics in the pipeline for Duchenne, PPMD has developed the Xcelerate program to fund projects at these stages. We recognize the importance to the entire Duchenne community to keep the Duchenne research pipeline full and robust. We are excited to begin receiving submissions and will keep the community posted on which projects are ultimately selected to receive these research funds. Learn more.
DuchenneConnect Receives $970,000 Grant
DuchenneConnect was recently awarded close to $1 million in funding from PCORI, the Patient-Centered Outcomes Research Institute and will be one of 29 members of PCORnet, the National Patient-Centered Clinical Research Network. The goal of PCORnet is to create a large, highly representative, national network for conducting clinical research. We are incredibly excited about this grant, and hope to ask and answer important questions about Duchenne and Becker muscular dystrophy using this huge, connected network. Learn more about this grant.
Stephen Groft, Champion of Rare Diseases Research, Retires
Steve Groft, PharmD led NIH's office of Orphan Products for 30+ years. He has been a champion for Duchenne, supporting our first step into advocacy in 2000 and sponsoring the first NIH workshop focused on Duchenne. His support continued over the years, providing advice and guidance about Standards of Care, registry development (DuchenneConnect), and research opportunities. He helped pave the way for relationships with NCATS and FDA. Congratulations Steve and thank you for all you have done to help us end Duchenne. You will be missed!
Nationwide Children's Hospital to Host Wellstone Center Conference
Join Nationwide Children's Hospital on Saturday, April 12 for the 3rd annual Wellstone Center Conference for patients and families. Held at the Education Center at Nationwide Children's Hospital, Columbus, Ohio the program features keynotes such as Updates on Exon Skipping and Gene Therapy by Dr. Jerry Mendell, Genetic Modifiers of Disease Severity in DMD: How Do We Explain Variation Between Boys by Dr. Kevin Flanigan, and Perspectives from an Electric Chair by Mo Gerhardt. Separate educational tracks for adults, teens, school age children and child care provided. Visit NationwideChildrens.org/edu to learn more.
Other Research News
The latest from Prosensa – Outlining next steps for Drisapersen
Download the latest PTC Therapeutics Webinar hosted by PPMD
Visit PPMD's Duchenne Research News Section for the latest Duchenne Headlines
Advocacy
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. We continue to demand that the FDA use all available tools given to them by Congress to approve rapidly treatments that show safety and effectiveness. Here are 3 simple actions you can take to help.
Action Alert: Ensuring Federal Agencies Working on Duchenne Receive Proper Funding
While the MD-CARE Act remains a top priority, we need to also make sure that federal agencies receive adequate funding and direction for the programs that are so critical to our community. One tool we use to ensure that funding and direction is our Congressional funding letter. The investment to date is over $250 million on Duchenne – let's keep adding to that number. It takes less than a minute to send your message to Congress.
Save the Orphan Drug Tax Credit Letter
A proposal made public by Representative Dave Camp (R-Michigan) to amend the Internal Revenue Code of 1986 would repeal the Orphan Drug Tax Credit, one of the most successful tax credits ever passed by Congress, and one that has literally saved thousands of lives. The credit is an incentive to companies developing therapies for rare diseases. PPMD joined The National Organization for Rare Disorders (NORD) and over 130 patient organizations in a letter to Congress strongly opposing this proposed repeal. Read the letter.
Sign Up for PPMD Action Alerts
Advocating for people with Duchenne is not just one month, it is year round. Sign up for PPMD's Action Alerts so that you and your family can take action when your voice is needed most.
Where it All Began
Our voices made a difference in 2001 – Adding years to the lifespan of individuals with Duchenne, investing millions of dollars in research, and moving potential therapies closer to approval. Our voices can make a difference again. Take a look back at the beginning of the MD-CARE Act and then do your part to make sure this historic legislation continues to get Congressional support.
Other Advocacy News
Mr. Stalling Goes to Washington! One young man's adventures in D.C.
Hoping for a Miracle: A Kennedy family is fighting for a new drug treatment that could save their son, and thousands like him
PPMD's advocacy strategy is multi-agency (FDA, NIH, CDC) and multi-layered. Download PPMD's webinar outlining our overarching advocacy strategy, laying out our goals for 2014, and discussing how you can advocate from home
Community
Registration Now Open for PPMD's 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 bring 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.

We can't wait to see you and your family in Chicago!

P.S. If you are a newly diagnosed family, attending the Connect Conference for the first time, please make sure you check out our support program.
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! Learn more.
Run For Our Sons
Upcoming Races
We've added two brand new races to our 2014 race calendar – the Kaiser Permanente Colfax Marathon in Denver and the DICK's Sporting Goods Pittsburgh Marathon. Join us in May for these exciting races and challenge yourselves on a whole new course. View our full race calendar.
Run For Our Sons Team Jackets Now Available
Show your team pride off the course with our new Run For Our Sons jackets! These super-stylish water-resistant, lightweight jackets will keep you dry and warm whether you're running on the course or cheering from the sidelines. Men's and Women's sizes available. Order yours today.
Now recruiting:
Hope4AJ 5K - March 29, 2014
2014 Unionville Run For Our Sons 5K - April 5, 2014
Dancing with the Spartans - April 11, 2014
Pins for Duchenne - April 12, 2014
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
Disneyland Half Marathon Weekend - August 29 – 31, 2014
Bank of America Chicago Marathon - October 12, 2014
Baltimore Running Festival - October 18, 2014
TCS New York City Marathon - November 2, 2014
And Save the Date for these popular 2015 races!
• Walt Disney World Marathon Weekend - January 8 – 11, 2015
• 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
For years our community has been making noise so that the scientific and medical communities would listen and focus on Duchenne. They're listening – now we need to talk! Filling out a survey or doing an interview about your family's experiences with Duchenne creates important data. This data is what clinicians, scientists, and government agencies need to develop new interventions, approve drugs, improve care, and ultimately help us end Duchenne. How can you add your voice?
Upcoming Events
• March 29, 2014: Hope4AJ 5K
• April 5, 2014: 2014 Unionville Run For Our Sons 5K
• April 11, 2014: Dancing with the Spartans
• April 12, 2014: Pins for 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
Propose research that is important to you
As Pat mentioned above, adding your experiences with Duchenne or Becker to ongoing research is vital to improve care and optimize potential therapies. But our community can go further. Let's set the research agenda! Tell us what you wish you knew (or your doctors knew, or therapists knew, or teachers knew, or researchers knew...) about Duchenne or Becker muscular dystrophy. Be as specific as possible. Share your ideas.
Nutrition, Energy, and Duchenne
During her years of clinical practice, our VP of Clinical Care, Kathi Kinnett was often asked, "Is there something that I can feed my child to help slow down, or stop, the effects of Duchenne?" The absolute answer is – we don't know. But a new article has endeavored to address this question Learn more about this latest study in nutrition.
We Must Understand Heart Health in Duchenne
Cardiac concerns continue to be a top priority for PPMD. One way we have taken action to further explore these heart issues is by joining together with the National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health (NIH) to develop a cardiac working group. Parent Advocate Brian Denger shares the story behind this partnership and how important this working group will prove to be.
Living Duchenne: The Alternative
There are care guidelines for improving both the quality and the quantity of life with Duchenne. But it isn't easy. There are always trade-offs. In her monthly blog Living Duchenne, Colorado/Wyoming FACES Coordinator Ivy Scherbarth describes the frustrating and upsetting conversations we often must have with our child who has Duchenne – but argues that these tough talks are better than the alternative. Read Ivy's blog and share with us your own feelings.
More Education Updates
To Tell or Not to Tell – That is the Question: Read the latest blog from Mary-Lou Weisman and share your thoughts with her
CDC's Muscular Dystrophy Surveillance Tracking and Research Network launches population-based muscular dystrophy tracking program in the United States. This program addresses gaps in public health research of muscular dystrophy
Avoiding the Emergency Room: How to Protect Your Child This Winter
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
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PPMD Community
DuchenneConnect
Run For Our Sons
Coach to Cure MD
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© Parent Project Muscular Dystrophy 2014 — All Rights Reserved.

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