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Parent Project Muscular Dystrophy
Join Our Team. Tackle Duchenne.
September 2017
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A week later and I am still overwhelmed by the incredible job our entire community did on September 7 for World Duchenne Awareness Day! My social media feed was dominated by families sharing their stories, their Duchenne journeys, along with amazing photos and videos.

All of September has been overwhelming! This community continues to work together to ensure the momentum we have going in research, care, and advocacy continues. And there’s plenty of September left for you to take action, including one of the highlights of the calendar year Coach To Cure MD on September 30! This football season marks the 10th anniversary of one of our most important awareness and fundraising events of the year. Check out our calendar full of daily actions.

PPMD is already starting to think about 2018 and just last weekend announced the states we will be visiting in the New Year as part of our End Duchenne Tour. Save the dates and stay tuned for more details.

Thank you for joining the fight and taking action this month. Let’s make the second half of September even more meaningful than the first!


Pat Furlong
PPMD Founding President & CEO

[Webinar Recording] PART 2: Gene Therapy for Duchenne - Nationwide Children's Hospital - September 2017
Last week, Dr. Jerry Mendell of Nationwide Children’s Hospital joined PPMD for a webinar to discuss his upcoming gene therapy trial, including trial design, inclusion/exclusion criteria, and timelines. If you missed it, you can now watch the recording of our webinar with Dr. Mendell.
FDA Announces Approval of First Gene Therapy in the United States
In late August, the FDA approved Kymriah, a gene therapy that treats an aggressive type of leukemia. For our Duchenne community on the brink of starting gene therapy clinical trials, this news is another piece of evidence showing the tremendous strides this technology has made, and reinforces PPMD's decision to heavily invest, with your help, in gene therapy through our Gene Transfer Initiative. With our $2.2 million grant to Dr. Jerry Mendell, Dr. Louise Rodino-Klapac (co-PIs), and Nationwide Children’s Hospital in January, the trial at Nationwide is making tremendous strides towards entering the clinic in the last quarter of 2017. PPMD is pleased for the patients that can benefit from Kymriah and optimistic about what gene therapy can do to help end Duchenne.
Roche Provides Community Update on BMS-986089 (RG6206)
Earlier this year, Bristol-Myers Squibb (BMS) announced it had entered into an agreement to license BMS-986089 (RG6206), an anti-myostatin adnectin in development for Duchenne, to Roche. Last week, the Roche team provided an update to our community outlining their transition plan with this therapy, including the progress of their ongoing recruitment for the BMS-986089/RG6206 trial. We appreciate both BMS and Roche’s transparency during this transition and look forward to continued updates on this trial.
Sarepta Therapeutics Announces Positive Results from 4053-101 Study
Sarepta Therapeutics announced muscle biopsy results from its 4053-101 study, a Phase 1/2 first-in-human study conducted in Europe to assess the safety, tolerability, pharmacokinetics, and efficacy of golodirsen (SRP-4053) in patients with confirmed deletions amenable to skipping exon 53. Sarepta reports that the study achieved statistical significance on all primary and secondary biological endpoints.
Other Research News
First Patients Enrolled in UK’s Early Access to Medicines Scheme for Santhera’s RaxoneŽ in Duchenne
Sarepta Therapeutics Quarterly Update: September 2017
Newly Published Data Validates Micro-Dystrophin Gene Therapy Approach in Animal Mode
Understanding Clinical Trials in Duchenne
New Test for Screening of Duchenne in Newborn Babies
Webinar Recording: Gene Therapy for Duchenne
2017 Connect Conference Resources & Recordings
PPMD Webinar Archive
Deadline to Submit Written Comments for Ataluren Ad Comm is Today
REMINDER: The deadline to submit a written comment for the FDA Advisory Committee Meeting on ataluren is today. To make a comment, view the notice document and click COMMENT NOW. Click here for some tips and things to think about when drafting remarks. Every voice matters in the Advisory Committee process make sure your voice is heard so that families have options!
MD Community Moves Forward ICD Code Nomination for Duchenne/Becker
The lack of an ICD code specific to Duchenne/Becker has proven a barrier to diagnosis, care, surveillance, research, and access. Today our global MD community has come together to move forward a nomination for greater specificity in coding. While there will ultimately be many benefits to the overall health system once we have greater coding specificity, it is our hope that a specific ICD code for Duchenne or Becker MD will allow for payers to better understand how investments into therapeutic interventions can impact cost utilization long-term. We are also eager to be able to assess how care standards are being implemented.
PPMD Lends Leadership to NCATS Rare Disease Research Toolkit Development
PPMD has had the distinct pleasure of serving as a part of a leadership team at NCATS who has worked collaboratively to create the NCATS Rare Disease Research Toolkit. Developed in partnership with patient community innovators, we convened patient community leaders with expertise in facilitating therapeutic development across the research continuum. Learn more.
FDA Reauthorization Act Signed into Law
Marking the end of an extensive two-year process involving patient organizations, FDA, industry, Congress, and the White House, the FDA Reauthorization Act was signed into law on Aug. 18. PPMD was proud to be an active participant to ensure that the new law reflected the needs of our Duchenne community and innovation opportunities within Duchenne. FDARA contains provisions to further enhance the patient-focused drug development efforts that build on PDUFA-V and the 21st Century Cures Act – both bills that have served as foundational to PPMD’s PFDD efforts and our ability to ensure that patient experience data is considered within product review. Learn more about this critical legislation that our community helped drive.
PPMD Says Goodbye to Mo Haider
Another extraordinary light has gone out in our community... We are deeply saddened to learn that Mo Haider has died at the age of 28. Mo has been an unwavering advocate for our Duchenne community on all levels for decades, most recently serving on PPMD’s Adult Advisory Committee. Through his participation in PPMD-led efforts such as the Draft Guidance for Industry and the development of the patient preference survey instrument, Mo inserted the critical perspective of the adult community. Our prayers and hearts go out to the Haider family. We are so grateful to have known Mo.
Other Advocacy News
The FDA Determination Letter and the Meeting Minutes from the Recent Pediatric Advisory Committee Meeting
The BENEFIT Act Passes the Senate
PDUFA VI Passes Senate
Senate Healthcare Bill Stalls
FY 18 Labor HHS Report Language Released
Local Advocacy Changing the World One Backyard at a Time
World Duchenne Awareness Day A Global Success!
On September 7, the global Duchenne community took action to help increase awareness and raise funds to end Duchenne, in support of the fourth annual World Duchenne Awareness Day and Duchenne Action Month. Cities issued proclamations, local landmarks lit up red, and thousands took to social media to talk about Duchenne. We were so moved by the stories, photos, and messages of hope that dominated our Facebook feed. Take a look back at the impact you made this year on World Duchenne Awareness Day!
World Duchenne Awareness Day: MVP
PPMD has been overwhelmed by the number of families, schools, communities, and companies that have participated in Duchenne Action Month so far this September! One family, though, has embraced numerous elements of Duchenne Action Month -- the Parzymieso Family! Last week, Susan and her family -- along with Christine Piacentino and her family -- hosted an incredible event in Western New York for World Duchenne Awareness Day! The celebration was also accompanied by proclamations from both the Mayor of the City of Buffalo and a NYS Assemblyman, as well as, a local news appearance to raise awareness about Duchenne. Hats off to the Parzymieso family for perfectly capturing the spirit of the day! And they’re not finished…later this month they will once again host a Coach To Cure MD fundraiser at their local high school. PPMD and the entire Duchenne community thank the Parzymieso Family for all they do to help end Duchenne!
It’s Not Too Late To Take Action!
September is almost half way over, but you still have time to take action! As you will see from our interactive Duchenne Action Month Calendar, there are a number of simple things you can do throughout September to make an impact in the fight to end Duchenne. Even if that day’s action is something you are already doing, encourage your friends and family to join the fight! Click here to open an expanded view of the calendar, and then click on each day to see how you can take action all month long!
Coach To Cure MD
Our 10th anniversary Coach To Cure MD is turning out to be our best ever. Over 500 schools with more than 9,800 coaches already registered including over 160 schools requesting families! Have you taken the survey to be part of our anniversary team or planned your tailgate to watch your favorite school play? There’s still time to help #TackleDuchenne and be a part of our 10th Anniversary.
Run For Our Sons
.1Ks Take Off!
Industry partners Sarepta Therapeutics and Mallinckrodt Pharmaceuticals celebrated Duchenne Action Month with employees participating in company .1Ks! Thank you to both companies for supporting the Duchenne community and helping raise awareness during this special month. If you’d like to host a .1K in your community, click here. It’s one of the easiest and most impactful events you can host!
A Magical Disneyland Weekend
Thank you to our incredible team of 53 runners, walkers, and rollers who represented Run For Our Sons at the Disneyland Half Marathon Weekend earlier this month. Our team members withstood a California heatwave and early morning start times to raise more than $107,000 to help end Duchenne! A special congratulations to Nick Archdeacon, Isaiah Mejia and Carter Londono for completing the 5K in their wheelchairs with the help of their moms! It was a special weekend for our community and we are so grateful to everyone who ran, fundraised and supported our team along the way! Check out some photos from the weekend on our Facebook page.
Bank of America Shamrock Shuffle
Kick off the running season in Chicago on March 25 at the Bank of America Shamrock Shuffle! You can choose from an 8K run or the 2-mile walk, so the whole family can participate. This race is a longstanding tradition in Chicago and for good reason! It’s fun, festive, and a great way to see the city by foot! Join the Run For Our Sons team and go the distance to end Duchenne this spring in Chicago!
Baltimore Running Festival
There’s still time to join our team for the Baltimore Running Festival on October 21st! This fun race series includes a 5K, half marathon, full marathon and full marathon relay along Baltimore’s scenic Inner Harbor and the city’s historic neighborhoods. It’s a great race you won’t want to miss! Sign up today.
Now recruiting:
Rock 'n' Roll Race Series - Nationwide, 2017 & 2018
GoodLife Fitness Victoria Marathon - October 8, 2017
Baltimore Running Festival - October 21, 2017
Rock 'n' Roll Savannah Marathon - November 4-5, 2017
TCS New York City Marathon - November 5, 2017
Walt Disney World Marathon Weekend - January 3-7, 2018
Chevron Houston Marathon & Aramco Houston Half Marathon - January 14, 2018
Bank of America Shamrock Shuffle - March 25, 2018
Upcoming Events
• September 23, 2017: PPMD Dream Dinner & Auction
• September 23, 2017: Coach To Cure MD No Label Brewing Co.
• September 24, 2017: Holy Mackerel Brewery Event to End Duchenne
• September 30, 2017: Peace, Love, Run 5K
• September 30, 2017: Miles for Matthew 5K/10K
• September 30, 2017: Coach To Cure MD
• October 8, 2017: Bank of America Chicago Marathon
• October 8, 2017: GoodLife Fitness Victoria Marathon
• October 14, 2017: PPMD's Every Single One Tour - Boston, MA
• October 14, 2017: Garrett’s Hero Run
• October 14, 2017: Argyle Run For Our Sons 5K/1M Fun Run
• October 19, 2017: Sam’s Night Texas
Check out our full Calendar and sync it with your personal calendar!
The 2018 End Duchenne Tour Coming to a City Near You!
Knowledge is power! PPMD is excited to announce next year’s lineup of areas we will visit as part of our End Duchenne Tour! Combining each of the pillars that make up PPMD’s mission, the End Duchenne Tour brings updates on research, advocacy, and care to cities across the country, featuring a roster of leading experts in the Duchenne space. Join us on the 2018 End Duchenne Tour!
Back-to-School & Talking to Teachers
Download PPMD’s Back-to-School Kit today so that you are armed with the resources you need to talk to your child’s school administrators and teachers. And if you use any of these resources, please share photos and feedback with us by emailing us!
PPMD’s Every Single One Tour Recap: Charleston, WV
We kicked off our fall Every Single One Tour stops in West Virginia this past weekend! Thank you to the families that attended, our speakers from Nationwide, James Poysky who made the trip from Houston, all of our industry presenters, and especially to the attendees from the nursing program at the University of Charleston that were a tremendous help throughout the day! We look forward to seeing you on the road as we head out to Boston, MA on October 14th, Albuquerque, NM on November 4th, and Pittsburgh, PA on November 4th!
Care Complications -- Going Beyond the More Common Elements of Care in Duchenne
At PPMD's 2017 Connect Conference, we featured a Care Complications Panel to discuss topics that are critical in the management of Duchenne -- Chest Pain, Flu and Airway Emergencies, Fat Embolism, and Bone Fractures. All of these topics are critical -- because all can be life threatening if not recognized and cared for appropriately. We hope that you will watch and re-watch these incredibly important presentations.
More Education Updates
PPMD's Bone Health Workshop Published
PPMD Launches Online Community Resource Center
Simple Actions
$15 off your next order!

Restock your family's supply of PPMD merchandise so that you can raise awareness this September as part of Duchenne Action Month! Shop now and save $15 on your order of $75 or more using promo code: SEPTEMBER!
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.
Parent Project Muscular Dystrophy
401 Hackensack Avenue, 9th Floor
Hackensack, NJ 07601
t. 800-714-5437
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© Parent Project Muscular Dystrophy 2017 – All Rights Reserved.

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