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Awarding Grants, Recognizing Progress,
Welcoming Leadership

July 2014
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The 20th Annual Connect Conference last month in Chicago was perhaps the best conference we have ever hosted, on a number of levels. Every year, more and more genius researchers and physicians and care providers attend. Every year, more and more pharmaceutical companies and biotechs attend representing growing interest in Duchenne. Every year, more and more families join us to connect – and not just mom and/or dad, but entire families including dozens of young people living with Duchenne. What started off 20 years ago as almost a lecture series of experts talking to parents, has become a dialogue with everyone interested in the perspective of everyone else in attendance. I'm so proud that PPMD can orchestrate these conversations. Read my recap blog, including a great video from the surprise flash mob!

It was also more than a conference in other ways. We submitted the first patient-advised draft guidance in rare disease to the FDA. We convened an incredible panel of physical and occupational therapists in our pre-conference meeting to discuss clinical trial outcomes. And we announced that John Porter, PhD will be joining the PPMD team. You can read more about all three of these announcements below.

It felt a bit like Christmas in June I must admit. I am still glowing from a great week with all of you. And for those of you who couldn't attend, I hope you'll start thinking now about joining us next June in Washington, D.C.

Enjoy summer!


Pat Furlong
PPMD Founder & President


Research
20th Annual Connect Conference
Awarding Grants, Recognizing Progress
At the Welcome Reception of last month's Connect Conference, we presented PTC Therapeutics with the Path to Progress Award in honor of their conditional approval from the EMA. We also announced that Prosensa will receive a $200,000 grant to fund the development of a multi exon-skipping strategy for Exons 10-30.
Welcoming Leadership
During the final session of the Connect Conference, Pat welcomed John Porter, PhD to the PPMD team effective January 2015. Dr. Porter is a Program Director at the NIH/NINDS, responsible for managing a portfolio of research grants across neuromuscular disorders. He has also long been a friend to our Duchenne community. After the announcement of his retirement, we asked John to join the PPMD team, to help us build upon current programs to expand the pipeline, accelerate progress, and approvals. We are thrilled that John said yes!
Conference Presentations Coming Soon
In between the Welcome Reception and the closing session, though, there was an extraordinary amount of valuable information shared by leaders in the Duchenne space from around the globe. If you are unable to attend the 20th Annual Connect Conference in Chicago, June 26-29, 2014 or if there is a session you would like to revisit, we will be posting presentations in the coming weeks.
HT-100 Receives Fast Track Designation from FDA
Akashi Therapeutics, Inc. (formerly DART/Halo Therapeutics) announced the presentation of preliminary positive clinical data for its most advanced drug candidate, HT-100 (delayed-release halofuginone), an orally available, small molecule developed to reduce fibrosis and inflammation and promote healthy muscle regeneration in boys with Duchenne. PPMD is proud to have been an early supporter of this research. Read the press release.
Other Research News
Sarepta Therapeutics Reports Long-Term Outcomes Through 144 Weeks from Phase IIb Study of Eteplirsen in Duchenne Muscular Dystrophy
Summit presents new data from Phase 1b clinical trial of SMT C1100 for treatment of Duchenne
Translarna (Ataluren), PTC Therapeutics' nonsense suppression drug has received Conditional Approval at the EMA, Europe's regulatory agency.
Prosensa Announces A Regulatory Path Forward for Drisapersen as a Potential Treatment for Duchenne
Two New Funding Opportunities from the Department of Defense's Duchenne Muscular Dystrophy Research Program. Learn more.
DuchenneConnect Annual Report: Registry Growth & Expanded Services
Advocacy
Strength Happens Together: PPMD Submits FDA Draft Guidance on Duchenne
June 25 was a landmark day for PPMD and the entire Duchenne community. After 2 decades of hard work and resolute persistence within the policy, legislative and regulatory arena we were able to provide to the US Food and Drug Administration the first-ever patient-initiated guidance to help accelerate development and review of potential therapies for Duchenne.

This guidance is a major milestone for the community and truly incorporates the patient voice – the community's voice – in well-documented and quantifiable ways in pursuit of our common goal – to end Duchenne.

We need to ensure that we do everything possible to speed the development of ALL therapies in the pipeline, at all stages of development, and better inform companies about clinical trial design. Learn more about this historic moment and what we can expect next.

Download the recording of our telebriefing held for the media on June 25 to learn more about the guidance, the 80+ expert stakeholders who contributed, and goals for the guidance.
One Step Closer
Progress! Yesterday the House Energy & Commerce committee voted unanimously to advance H.R. 594, the Paul D. Wellstone Muscular Dystrophy Community Assistance, Research and Education Amendments of 2014. This action moves the bill to consideration and hopefully passage by the full House of Representatives. PPMD has championed for co-sponsors for more than a year and to date the bill is cosponsored by more than 100 members of the House of Representatives! PPMD appreciates the work of many offices, particularly that of our sponsors – Rep. Michael Burgess of Texas and Eliot Engel of New York – that has gotten us to this stage. But what's next?
PPMD President Pat Furlong Testifies Before Key Congressional Committee
PPMD President Pat Furlong was chosen as one of four patient-advocacy organization leaders to participate in the House Energy and Commerce Committee hearing entitled "21st Century Cures: Incorporating the Patient Perspective" on July 11, 2014. She used this opportunity to address the MD-CARE Act, the recent submission of a draft guidance to the FDA, and PPMD's benefit/risk project. We are hopeful that continued public and private collaboration, like the actions led by PPMD and the new ideas generated by the 21st Century Cures Initiative, will achieve even more to help end Duchenne and so many other serious diseases. Read the press release about Pat's participation in the hearing.
Senator Dick Durbin Named "Change It Champion"
PPMD was honored to be joined by a very special guest at our 20th Annual Connect Conference, 2014 "Change it Champion" award recipient Senator Dick Durbin. We deeply appreciate Senator Durbin's efforts to advance the American Cures Act, which would benefit the Duchenne community immensely. We are also grateful for his support for NIH, CDC, and FDA investment in Duchenne research, public health, and access strategies. Thank you Senator!
Other Advocacy News
PPMD Southern California FACES Coordinator, Mindy Cameron, shares her Duchenne journey with Department of Defense Duchenne Muscular Dystrophy Research Program
PPMD Benefit/Risk Study Results Published in Clinical Therapeutics
The State of Illinois recognized our 20th Annual Connect Conference in Chicago, IL, June 26-29 by declaring the week of June 23 as Duchenne Muscular Dystrophy Awareness Week!
Community
Help Us Tackle Duchenne!
We know it's hard to think about the fall when summer is heating up and underway. But now's the time to start planning your Coach To Cure MD event! One of the most successful, national awareness campaigns in the Duchenne community, Coach To Cure MD, is a great way for you and your family – and your entire community! – to raise awareness and funds that help us end Duchenne. PPMD provides you all the tools you need to host a great local event. You just need to reach out. Email our team leader, Danielle, and she'll get you started. It's time to join our team and help tackle Duchenne!
Run For Our Sons
Run the Big Apple to End Duchenne
We have a handful of entries left for the TCS New York City Marathon, one of the largest and most sought-after races in the country! You'll explore all 5 boroughs before a triumphant finish in Central Park. Join our team and wear our green to help end Duchenne. Register today.
Choose your Running Destiny!
There's a run for Jedi Knights and Padawans of every age and skill level during the Inaugural Star Wars Half Marathon Weekend at Disneyland, including the Star Wars Half Marathon, Star Wars 10K and Star Wars 5K. Or put yourself to the ultimate test and join the Star Wars Rebel Challenge combining the Star Wars 10K and Half Marathon for an out-of-this-world running adventure! Join us in a galaxy far, far away January 15-18, 2015.
Run Through History
Tour one of America's most historic cities, Philadelphia, in a whole new way by participating in the Rock 'n' Roll Philadelphia Half Marathon & 5K in September. You'll pass by the Liberty Bell, Liberty Hall, and lots of other historic sites and then celebrate at a post-race festival headlined by Rusted Root. Grab some friends, join our team and enjoy a fast, flat race course while helping us end Duchenne.
We Rocked Seattle!
Thank you to all of our runners, walkers, and rollers, who participated in June's Rock 'n' Roll Seattle Marathon & Half Marathon. Together they raised nearly $5,000 and had a great time doing it!
Now recruiting:
Rock 'n' Roll Philadelphia Half Marathon & 5K - September 20 – 21, 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
Star Wars Half Marathon Weekend - January 15 – 18, 2015
Chevron Houston Marathon & Aramco Houston Half Marathon - January 18, 2015
Rock 'n' Roll New Orleans Marathon - January 25, 2015
Simple Actions Have Lasting Impact
Everyone loves a clever sales pitch. And coming up with something simple, short, and clever is the best way for you to turn your Duchenne fundraising idea into a successful reality! Sometimes just a simple lemonade stand, with a great tagline can change hearts and raise money.

Thanks to the "PPMD Lemonade Divas" for kicking off summer with their lemonade stand to raise money for Sean's Dream Team's Disney World Marathon fundraising efforts. Meeting your fundraising goal for Run For Our Sons doesn't just have to come from direct donations. And not everyone can host a large gala or silent auction. Sometimes it just takes a simple action, some creativity, and an investment of a couple of bucks. Contact PPMD today with your great fundraising idea and we'll give you the tools and support you need to make it a success! Oh, and their tagline? "Squeezing out Duchenne with sweetness and a smile!"
Upcoming Events
• July 26, 2014: 30th Annual Friar Fight Golf Tournament to End Duchenne
• July 27, 2014: The San Francisco Marathon
• August 16, 2014: Mitchell's Run
• August 29-31, 2014: Disneyland Half Marathon Weekend
Check out our full Calendar and sync it with your personal calendar!
Education
PPMD and NHLBI Co-Sponsor Duchenne Cardiac Working Group
PPMD continues to make cardiac issues – which affect ALL people with Duchenne – a priority. We are thrilled to partner with the National Heart, Lung, and Blood Institute (NHLBI) to convene a working group titled "Contemporary Cardiac Issues in Duchenne Muscular Dystrophy." The group, made up of top pediatric and adult cardiologists as well as clinical researchers, met last week to address critical evidence gaps in the understanding of cardiac issues in Duchenne. Thanks to John Owens Adventure, Inc. for their financial support of this endeavor! A formal executive summary will be forthcoming from NHLBI, but you can read a brief summary here.
Outcomes Meeting: Laying a Foundation for Outcome Measures
PPMD convened a pre-conference meeting of physical therapists, occupational therapists, and clinical coordinators who specialize in the research, assessment, longitudinal data collection, and treatment of people with Duchenne. The Outcomes Meeting gave both therapists and industry a forum for discussing the standardization of the physical therapy evaluation of a child with Duchenne. Find out about what was discussed at this meeting and next steps.
PPMD Names Children's Hospital Colorado a Certified Duchenne Care Center
Children's Colorado has been a pivotal partner with PPMD in the Transforming Duchenne Care Initiative, along with Nationwide Children's Hospital in Columbus, Ohio, which is the only other recipient of the Certified Duchenne Care Center Program. Dennis J. Matthews, MD, a neuromuscular specialist and chair of Pediatric Rehabilitation Medicine at Children's Colorado, has played an integral part in the TDCI. As a noted expert in Duchenne care, Dr. Matthews was involved in developing the measures now being used to evaluate neuromuscular care across the country. Read more about PPMD's Certified Duchenne Care Center Program.
International Study on the Burden of Duchenne Published in Neurology
In the first international study of its kind, researchers have found that there are many different costs accompanying a rare condition such as Duchenne muscular dystrophy and that there is a considerable financial burden carried by affected families. Funded by GlaxoSmithKline (GSK), the aim of this study was to estimate the total cost of illness and the economic burden of Duchenne. Thank you to all the DuchenneConnect families who participated in this important study! Continue reading.
Independence
How do you nurture your child's independence? It's a fine balance when raising a child with Duchenne – finding the perfect combination of giving him the support he needs, but also helping him find his way. Colorado/Wyoming FACES coordinator and Duchenne mom Ivy Scherbarth shares her thoughts about independence in her latest blog. We'd also love to hear from you. Join the conversation on our community site. Read Ivy's blog.
More Education Updates
Good-bye: An open letter from Mary-Lou Weisman, saying goodbye to her blog after two incredible years of sharing her insights, stories, and heart.
EZ Lap Tray is a revolutionary new design for people of all ages, including those with physical disabilities or limited mobility. The tray provides comfort, stability, and independence.
Education, Behavior, and Duchenne: PPMD FACE Coordinator and Education & Behavior Specialist, Jill Castle, has launched a blog for families to provide behavioral and education support.
Simple Actions
Want to Win a $25 Amazon Gift Card?

If so, update your DuchenneConnect account to be entered into a monthly raffle! On the last day of each month of this year, we will draw one winner from the accounts that have been updated that month.

We will also draw a winner from all the new registrants each month, so please encourage any relatives or friends who have Duchenne or Becker to register today. Remember that the registry is not just for patients affected with Duchenne or Becker, but also for carrier females.

All of this work has been made possible by a funding award DuchenneConnect received from the Patient-Centered Outcomes Research Institute (PCORI). We were selected to be one of 29 networks in PCORnet, a large health data network. This exciting news means that DuchenneConnect, already a model for rare disease registries, can grow to the next level. We are extremely enthusiastic about maturing the registry with input and support from our collaborators, the PCORnet partners, and YOU!

Email Centered Outcomes Research Institute (PCORI). Email coordinator@duchenneconnect.org with questions.
Free Genetic Testing Program

PPMD and DuchenneConnect, a program of Parent Project Muscular Dystrophy, have launched 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.
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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Run For Our Sons
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© Parent Project Muscular Dystrophy 2014 — All Rights Reserved.

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