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Time to Connect
June 2014
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This is always one of the busiest and most exciting times of year for PPMD. We are just weeks away from what promises to be our most incredible, comprehensive Connect Conference to date. What an appropriate way to mark 20 years in the fight to end Duchenne!

If you've been to the Connect Conference before, you know that this is your opportunity to hear from the top minds in the Duchenne space. You have access to scientists, researchers, physicians, care providers, and industry – people who have dedicated their professional life to helping your child.

It's also about connecting with each other. I am always amazed at the amount of talent and the progress being made in Duchenne. But what excites me most about the conference, year after year, is seeing you and your family. Meeting families, watching kids interact and be kids, catching up with old friends – it's the highlight of my year and I hope to see you there!


Pat Furlong
PPMD Founder & President


Research
Streaming Available for the 20th Annual Connect Conference
If you are unable to attend the 20th Annual Connect Conference in Chicago, June 26-29, 2014, we hope you will take advantage of our live streaming of most sessions. This year's agenda provides the latest research updates from the leaders behind all the exciting projects in the therapeutic pipeline. Information on how to stream will be shared on our website, the community site, and Facebook when the Conference begins June 26.

Many of the presentations will be shared on our website too, in the weeks following the Conference. Registration is open if you still want to attend though.

Don't let distance keep you from critical information.
Catena®/Raxone® Positive Results Reported from Phase III Duchenne Trial
Santhera reported positive results from Catena®/Raxone® in their Phase III Duchenne trial. These results are supported by additional respiratory function data. By including pulmonary outcomes in a study like this, we hope other companies are encouraged to include adult (non-ambulant) patients in their clinical trials. Ideally this would lead to broad labels and indications that enable every patient access. Read the release.
PTC Therapeutics Receives Positive Opinion from CHMP for Translarna (Ataluren)
Ataluren, PTC Therapeutics' nonsense suppression drug has received Conditional Approval at the EMA, Europe's regulatory agency. This is a big moment for Duchenne, a landmark decision, and a sign of hope for all. While ataluren is targeted at a small subset of patients (13% +/-), the conditional approval is a sign that the EMA worked very hard to understand Duchenne, that they recognize this unmet need, the progressive and debilitating process of Duchenne, and the urgent need to treat people with Duchenne. Learn more and read the release.

Watch PTC's recent webinar regarding the news out of EMA.
Prosensa Announces A Regulatory Path Forward for Drisapersen as Potential Treatment for Duchenne
Prosensa announced plans to submit a New Drug Application to the FDA later this year and continues their dialogue with the EMA with the intent to seek approval from the European agency as well. This news from Prosensa continues to demonstrate that FDA recognizes the urgent needs of this community, the importance of the patient voice to understand the benefit/risk assessment, and continues to demonstrate flexibility in the review process. Read the release.
SMT C1100 Preliminary Results from Phase 1B Clinical Trial for Treatment of Duchenne
In the first study of a utrophin up-regulating drug in Duchenne, British biotech company Summit PLC reports that its drug SMT C1100 showed a good safety profile in twelve boys with Duchenne. Plasma levels of the drug were variable and the company speculates that diet and the course of the disease may be the cause. The preliminary trial data will be reviewed further by Summit and is expected to lead to a revision of future clinical trial plans in order to determine the optimal way, either through dietary means or drug formulation changes, to address the drug uptake differences between Duchenne patients and healthy volunteers. The next patient study is now expected to start in Q4 2014. Read more.

Watch Summit's recent webinar regarding the Summit report.
DART Therapeutics Continues Clinical Development of HT-100 for Duchenne
DART Therapeutics Inc. announced that it has resumed clinical development for its lead drug candidate, HT-100 (delayed-release halofuginone) an orally available, small molecule drug candidate intended to reduce fibrosis and inflammation and promote healthy muscle regeneration in boys with Duchenne. The company will continue to enroll and dose Duchenne patients in this ongoing phase 1b/2a clinical study to evaluate the safety and tolerability of increasing doses of HT-100, and explore trends in a range of efficacy endpoints. Read more.
Other Research News
Two New Funding Opportunities from the Department of Defense's Duchenne Muscular Dystrophy Research Program. Learn more.
New Duchenne Therapies in Development – A Blog from PPMD's Vice President of Research, Sharon Hesterlee, PhD
Results of PPMD-Funded Study Prompt Phase III Study of Tadalafil in Duchenne
Sarepta Therapeutics Announces Plans to Submit NDA
DuchenneConnect Annual Report: Registry Growth & Expanded Services
Advocacy
Major Advocacy Milestone Marked
For the first time ever, the appropriations bill that funds the FDA includes report language focused on Duchenne! This is a significant milestone for the Duchenne community that has worked so hard over the years on both the benefit/risk project and draft guidance that we will soon be submitting to FDA for review. We thank Congresswoman Doris Matsui and Congressman Peter King for championing this language, and we thank the many members of Congress who lent their support! Learn more.
Another Victory for Duchenne Awareness in Congress
On May 20, the House Energy and Commerce Subcommittee on Health held a hearing entitled, 21st Century Cures: The President's Council of Advisors on Science and Technology (PCAST) Report on Drug Innovation. During the hearing, PPMD Champions and lead sponsors of the MD-CARE Act, Representatives Engel and Burgess, mentioned the importance of the MD-CARE Act, the impact it has had on potential therapies and the care of patients and stressed the need to utilize the expedited programs within the FDA, programs like accelerated approval and the "breakthrough therapy" designation. Watch the hearing.

We are being heard but now is not the time to stop using our voice. We must get the MD-CARE Act passed – it is more important than ever to keep federal support in the Duchenne space. Learn more.
99 Co-Sponsors of the MD-CARE Act in the House! Is Yours on the List?
Our 99th MD-CAREAct cosponsor in the House of Representatives! GREAT JOB to all our New Jersey families and friends who contacted Congressman Chris Smith [NJ-4] and asked him to sign on to the bill. If your Members of Congress have not yet taken action to Pass the MD-CARE Act, ask them to do so today!
Time to Deliver: Update on Draft Guidance for FDA
As you probably know, for the past four months over 80 experts in Duchenne (including patient representatives and groups, industry, clinicians, and researchers) have been working hard to produce draft guidance on Duchenne that we believe will ultimately speed the development of therapies. Earlier this month, you were given the opportunity to share your thoughts on the guidance content, so we can make sure we provide the FDA with a guidance that truly reflects the concerns and priorities of the ENTIRE Duchenne community.

Next week, PPMD will deliver the completed draft guidance to all of the FDA official who attended our Duchenne Policy Forum in December when the community was charged with drafting this historic document. This is one of our finest illustrations of "Strength Happens Together" and we couldn't be more pleased with the end result. Stay tuned for more about this major moment in Duchenne history.
Pat Furlong Receives FDA Honor
Congratulations to PPMD President & CEO Pat Furlong who is a recipient of the Group Recognition Award at the 54th Annual FDA Honor Awards Ceremony! This award recognizes superior achievement of the Agency's mission through teamwork, partnership, shared responsibility, and fostering collaboration and coalition to achieve FDA goals. Pat is part of a panel of experts who participated in the FDA's Patient Network Meeting: Demystifying FDA. We are so proud to be led by this extraordinary woman!
PPMD-led Treatment Preferences and Risk Tolerance Study Published
PPMD is thrilled that results from the recent benefit/risk project our team led have been published in Clinical Therapeutics. The article concludes that caregivers are willing to accept a serious risk when balanced with noncurative slowing or stopping of the progression of muscle weakness, even absent improvement in lifespan. These preferences should inform the Food and Drug Administration's (FDA's) benefit-risk assessment of emerging Duchenne therapies. Learn more.
"Right to Try" State Model Legislation
The Goldwater Institute of Arizona has designed state model legislation, known as a "Right to Try" bill, which would make experimental drugs available to terminally-ill patients without the FDA's approval. Under this legislation, a patient with "an advanced stage of a disease with an unfavorable prognosis and no known cure" could obtain an experimental drug that has passed initial toxicity and dosage testing (Phase I clinical trial) with the permission of their physician and the drug manufacturer. Learn more.
Other Advocacy News
We are thrilled that PPMD's Founding President & CEO Pat Furlong will represent the Duchenne community on FasterCures Benefit-Risk Advisory Council. Meet the Council.
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.

TRAVEL TIP: Open Taxis is a centralized dispatch for all Chicago wheelchair accessible vehicles (WAV). You can call 24/7 to request a WAV cab for immediate pickup or to pre-arrange a trip at 1-855-WAV-1010 (1-855-928-1010). More information is available at OpenTaxis.com.

Not registered to attend the Conference yet? Registration is still open!
Run For Our Sons
May the Course Be With You!
Join us in a galaxy far, far away for the Inaugural Star Wars Half Marathon Weekend! Find the force within you to run the half marathon, 10K, 5K or the Rebel Challenge (half marathon and 10K on back-to-back days) at Disneyland January 15-18, 2015. We hope to see you there!
Deep in the Heart of Texas
We're heading to Texas once again for the Chevron Houston Marathon & Aramco Houston Half Marathon on January 18, 2015. This has grown into one of the country's most popular marathons and you can skip the selective lottery process by joining the Run For Our Sons team! Get more info and register on our website.
Thanks to our May Racers
A big thank you to everyone who participated in a race on behalf of Run For Our Sons in May. We had teams all over the country, from Pittsburgh to Denver, running marathons, 10Ks and everything in between, and raising tens of thousands of dollar to help end Duchenne. View all of our race recaps.
Now recruiting:
Rock 'n' Roll Seattle Marathon & Half Marathon - June 21, 2014
The 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
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
Other Community News
One More Reason to Register for PPMD's 20th Connect Conference – A Great Party!
Simple Actions Have Lasting Impact
This time of year marks so many rites of passage for our kids, namely graduation. Whether moving up from Pre-K to K, high school to college, or law school to law firm, family and friends spend hours sitting in uncomfortable chairs grinning from ear to ear, wiping away tears as our babies move on to the next chapter of their lives. (Yes, even a twenty-something year old graduate is still his parents' baby!)

This month, we ask you to share with us pictures and thoughts you have about the graduate in your life, who in spite of Duchenne, has accomplished something huge and is moving up. Any age, any graduation – let's take a moment to congratulate these incredible young people who worked so hard for that diploma!

Congratulations Class of 2014! We are so proud of how you represent the Duchenne community out in the world!
Upcoming Events
• June 21, 2014: Rock 'n' Roll Seattle Marathon & Half Marathon
• June 26-29, 2014: 2014 Annual Connect Conference
• June 28, 2014: Cruising for a Miracle Poker Run
• June 28, 2014: Team Aiden Family Fun 5K Run/Walk
• 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!
Connect Conference: Register Now!
2014 Connect Conference in Chicago
Date: June 26-29, 2014
Location: Chicago Marriott Downtown Magnificent Mile
Register now.
Education
Good-bye: An Open Letter
Mary-Lou Weisman, the beloved author's of this community's must read memoir Intensive Care: A Family Love Story says goodbye to her blog after two incredible years of sharing her insights, stories, and heart. We will miss hearing from this teacher and friend on a regular basis, but she's only an email away! Visit her website to read her final blog and if Mary-Lou has helped you navigate this Duchenne journey we are on, take a minute to send her a note of thanks.
Pulmonary Care and Duchenne
Recently, Dr. Lisa Wolf gave a wonderful presentation at Duchenne Foundation Australia's Melbourne Symposium. While her presentation was titled, "DMD Respiratory Review 2014: The Younger Years," she was able to cover a wide range of respiratory topics that span the life of a person living with Duchenne. Learn more.
ENMC Workshop on Adults with Duchenne
We don't know a lot about adults living with Duchenne: their natural history, cardiac progression on and off steroids, on an off cardiac medications, the effects of other medications and/or supplements, endocrine issues, gastrointestinal issues, urologic issues, gaining and maintaining independence...the list could go on and on. Who would have dreamed that one day we would need to have a workshop on how to care for adults living with Duchenne?! What a wonderful problem to have! But for the hundreds of men and women living with Duchenne, these issues do indeed cause problems.

On May 23-25, over 30 experts from Europe and North America, including 2 young men living with Duchenne, gathered at the 206th European Neuromuscular Centre (ENMC) Conference to discuss issues of importance in the care of adults with Duchenne. Read a summary of the fabulous meeting that resulted in some very productive outcomes.
Healthy Summer Snacking
It's summer – finally! Growing children are hungry children, and hungry children don't usually go for the healthy snacks first. As parents, we try everything we can to get them to eat right: reasoning (this sometimes works), bribery (generally referred to as "creative parenting"), and sneaking nutrients into the foods they love (referred to as, well, just being sneaky). We all have tricks and we would love to hear some of yours! Send us your recipes for yummy, healthy snacks, and we will share them with everyone.
More Education Updates
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.
A New Care Guide to Give to Your Provider – Imperatives for Duchenne Care
Certain Medicaid Waiver Payments May Be Excludable From Income
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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