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Tackling Duchenne from Every Angle
November 2015
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When I look at a newsletter like this one and see all that's happening in our community I am overwhelmed by the momentum, the progress, the promise. Our industry partners continue to move forward with potential therapies, as new companies enter the market. Our efforts in Washington, both on Capitol Hill and with regulatory agencies continue to make strides in educating decision makers and sharing our stories. We are educating care providers on the latest tools and techniques to ensure people with Duchenne get optimal care. And you, our wonderful community of families, are going above and beyond to raise awareness and funds through incredibly creative grassroots events and by participating in long distance races all around the country.

If you look at social media this month, a lot of people are sharing what they're grateful. Like you, I have my list – my husband, my daughters, my perfect granddaughter. But there's also YOU and the integral role you play in our community.

A few years ago, you might remember PPMD had a t-shirt with the quote, "No one can do everything. But everyone can do something." A comprehensive approach that incorporates the talents of this entire community, working together...that's how we'll do it. That's how we'll end Duchenne.

With gratitude,


Pat Furlong
PPMD Founder & President


Research
PPMD Hosts Myostatin Inhibition Webinar
On Wednesday, November 11th PPMD hosted a webinar about the importance of myostatin inhibition research. Sponsored by educational partners Bristol-Myers Squibb and Pfizer, this webinar helped to put context and clarity around this treatment pathway so families have a better understanding as they see clinical trials in this area. Some years ago, researchers identified the myostatin pathway as a negative regulator of muscle growth. They isolated the cause of this disparity to a mutation in the gene that codes for the production of a hormone called myostatin, which tends to limit muscle growth. Scientists searching for a treatment theorize that inhibiting myostatin in boys with Duchenne will cause them to develop more muscle mass initially. Ideally, this surplus will offset the muscle loss associated with Duchenne, allowing boys to retain their ability to function for a longer period of time. Watch the latest in PPMD’s Webinar Series.
Understanding Decode Duchenne
The recording of our webinar about Decode Duchenne program is now available! Decode Duchenne provides FREE genetic testing to those who meet the criteria. We want to again thank Dr. Stan Nelson with the Center for Duchenne Muscular Dystrophy at UCLA and Kimberly Powers for being a part of this webinar. We are also very thankful for the support from BioMarin Pharmaceuticals Inc., PTC Therapeutics, and Sarepta Therapeutics. Their support is what makes this incredible program possible for our community! Click here to watch the webinar and learn more about the launch of Decode Duchenne by clicking here.
A Paradigm Shift in Drug Discovery
Your voice has made an important difference in how Duchenne is understood and prioritized by clinicians, drug developers, and regulatory agencies. The Critical Path Hill Briefing that was held last month demonstrated the importance of moving from “if and when [scientists and companies] are willing to share data” to an “institutionalized sharing of data.” It gives us better designed clinical trials, and allows their successful launch and touchdown at approval and marketing of effective drugs. It’s a new paradigm to embrace. Read more about this shift in drug discovery that PPMD is proud to help lead. And click here for a great video to understand what C-PATH does.
What's Next for Venture Philanthropy?
Venture philanthropies are emerging as strong and strategic partners in the medical research system. Five research leaders, including PPMD’s Annie Kennedy, discussed "What's next for venture philanthropy?" at Partnering for Cures, including how to define venture philanthropy and how foundations prioritize among the competing opportunities to invest their time and resources. Watch the presentation and read a summary of the panel discussion.
Other Research News
Santhera Reports New Data and Updates on Regulatory Filings for RaxoneŽ (idebenone) in Duchenne
European Commission Grants Orphan Medicinal Product Designation for CAT-1004, Catabasis Pharmaceuticals' Investigational Therapy for the Treatment of Duchenne
PTC Announces Results from Phase 3 ACT DMD Clinical Trial of Translarna™ (ataluren) in Patients with Duchenne
PPMD Launches Next Phase of Genetic Testing Program, Decode Duchenne in collaboration with BioMarin Pharmaceutical, PTC Therapeutics, & Sarepta Therapeutics
PPMD's PRO Project: Adding more tools to help with upcoming clinical trials in Duchenne
Advocacy
PPMD Submits Results of Patient-Centered Benefit-Risk Assessment Study in Duchenne & Becker to FDA
PPMD is pleased to announce that late yesterday we submitted the results of our most recent patient-centered benefit-risk assessment study to the FDA. Overall, using multiple approaches, the study demonstrates that patients and caregivers value pulmonary benefits and are willing to accept risks and burden to achieve those benefits. The assessment was done with research partners at the Johns Hopkins Bloomberg School of Public Health. Based on this study, which was sponsored by Santhera Pharmaceuticals, we are hopeful the FDA will have a better understanding of preferences of Duchenne patients and caregivers in regards to a specific pulmonary therapeutic target. Learn more.
What to Expect from the FDA Ad Comms
Earlier today, PPMD President Pat Furlong and Dr. Tim Franson, Chief Medical Officer of Your Encore and former VP of Global Regulatory Affairs and Drug Safety at Eli Lilly, discussed what we can expect out of the upcoming Advisory Committee meetings with the FDA. Not only did Pat and Tim discuss the kinds of recommendations that can come out of these meetings, but they examined how these recommendations potentially affect the FDA's ultimate decision on whether or not to approve potential therapies. We will link to today's webinar later this week, but in the interim click here to read more about what we have learned about this process.
PPMD Supports Advancing Targeted Therapies for Rare Diseases Act of 2015
For over a year, PPMD has been working with congressional champions and industry partners to help lead an effort to streamlining the regulatory pathways and review processes for targeted therapeutics (which would include such things as ‘follow on exons’) by clarifying the FDA’s existing authority to leverage data previously used in the approval of a targeted product when approving a new therapy. For that reason, we collaborated with the National Organization for Rare Disorders (NORD), the Muscular Dystrophy Association (MDA), the Duchenne Alliance, and many others to demonstrate the value of this bill and the power of our rare disease community. Please read the letter that PPMD is proud to be a signer on.
Help Us Protect Your Wheelchair Technology Access – Act Now to Close a CMS Loophole!
The Centers for Medicare and Medicaid Services (CMS) has announced that it will apply Medicare competitive bid program pricing to Complex Rehab wheelchair accessories effective January 1, 2016; an action that will likely reduce access to wheelchair features many of our Duchenne families depend on. We urge all members of our community to take action by urging your members of Congress to support H.R. 3229 in the House and S. 2196 in the Senate. Click here to take action.
Other Advocacy News
FDA Advisory Committee Meetings: An Overview of the Process & Ways to be Involved
Ad Comms Update: Group Letter Submitted to FDA
The Ensuring Access to Clinical Trials Act (EACT) Passes Senate, then House
Industry partners receive FDA's Pediatric Rare Disease Designation – and YOU helped make it possible
Community
Community Corner

School Spirit!
We love stories like this! Thank you to the staff at Matthew Paterson Elementary School for raising awareness and supporting our community's fight to end Duchenne!
"How amazing are my friends/colleagues at MPES!!!!? Look at them! They amaze me more and more with their love and support. This is how they welcomed me back to work today. Everyone had End Duchenne shirts on and will spread awareness all day now! I love you all so much!!!" -- Jessica Curran

Coach To Cure MD
When you hear the words Coach To Cure MD, you may immediately think football, but two high school teams in Missouri have brought it to the soccer field for the past three years. T.J. Clime and Mark McDonald were the honorary captains. T.J., a Jefferson City Jay and Mark, a Helias Crusader. Thank you to both teams for helping end Duchenne. Check out the photos from this year's event!

Run For Our Sons
Time is Running Out!
We have a handful of entries left for the Walt Disney World Marathon Weekend at Disney World and the Star Wars Half Marathon Weekend at Disneyland. Don’t miss these magical and fun-filled January race weekends, some of the most fun events on our race calendar!
Bank of America Shamrock Shuffle 8K
Hey Chicagoland runners! We’ve added the popular Bank of America Shamrock Shuffle 8K to our race calendar and we’d love you to join us! Kick off the spring running season with a one-of-a-kind course that weaves through Chicago’s Loop, starting and finishing in Grant Park.
Register today for this April 3rd race.
Bike to End Duchenne
We’re biking the Big Apple once again this year at the TD 5 Boro Bike Tour on Sunday, May 1st. See NYC like you’ve never seen it, riding it’s famous highways and major avenues with no traffic, and then celebrate at a fantastic finish line festival in downtown Manhattan!
Join 32,000 fellow bikers in this unique event, and ride the distance to end Duchenne.
Marin County Half Marathon
We’re excited to add the Marin County Half Marathon, 10K & 5K to our race calendar this spring! Choose from three race distances and enjoy the beautiful McNear’s Beach County Park in San Rafael, California. There’s even a free kids fun run the morning of the race so bring the whole family and enjoy a great day with us as we go the distance to end Duchenne.
Now recruiting:
Walt Disney World Marathon Weekend - January 7-11, 2015
Star Wars Half Marathon Weekend - January 14-17, 2016
Chevron Houston Marathon & Aramco Houston Half Marathon - January 17, 2016
Miami Marathon & Half Marathon - January 24, 2016
Rock ‘n' Roll New Orleans Marathon & Half Marathon - February 28, 2016
Bank of America Shamrock Shuffle 8K - April 3, 2016
Marin County Half Marathon, 10K & 5K - April 24, 2016
TD 5 Boro Bike Tour - May 1, 2016
Save the date:
• Star Wars Half Marathon - The Dark Side - April 14-17, 2016
• Million Dollar Bike Ride - May 7, 2016
Upcoming Events
• November 25, 2015: Hustle For Muscle 5K
• November 26, 2015: Turkey Trot to End Duchenne
• December 5, 2015: Creekside's First Annual Grant's Gang 5K Run/Walk
• December 5, 2015: Dance To End Duchenne
Check out our full Calendar and sync it with your personal calendar!
Education
DuchenneConnect Launches Awareness Video
You have the power to make a difference! Check out our new video about PPMD’s DuchenneConnect to learn why it is so important that we have as many families join the registry as possible. And if you already are registered with DuchenneConnect, share this with others in our community who may not know about this critical resource.
Win an iPad!
Speaking of DuchenneConnect, complete one of our surveys at DuchenneConnect.org and your name will be placed in our monthly drawing. You will notice that when you log in, we have recently made many more surveys available. Some of the ones you see may be partially completed with any data you have given us previously. If you have any surveys that are partially completed we ask that you complete those surveys to make sure all our data is complete and up to date. Answer a survey and you could win a free iPad!
Train the Trainers!
Last month, PPMD hosted back to back "Train the Trainers" sessions in upstate New York and Florida. These well-attended sessions helped parents and PTs learn special physical therapy techniques specific to people with Duchenne. Stay tuned for announcements for additional cities/dates in 2016.
Important Survey on Clinical Care

Are you or your child seen at a Certified Duchenne Care Center? If so, please take 15 minutes and tell us about the care you received. This survey is anonymous and the results are shared with the centers in aggregate so that they can improve the care they provide. Thank you for helping us continue to sustain and improve the care and services provided to you and your family!

More Education Updates
Update of the Duchenne Care Considerations
Revised Duchenne Anesthesia Recommendations for 2015
Updated Vaccination Recommendations
Simple Actions
Free genetic testing, interpretation, & counseling for people with Duchenne or Becker muscular dystrophy
Free genetic testing, interpretation and counseling for individuals with Duchenne or Becker muscular dystrophy. Decode Duchenne is administered by PPMD through DuchenneConnect and is funded by BioMarin Pharmaceutical, PTC Therapeutics and Sarepta Therapeutics. Visit DuchenneConnect.org today to learn more!
Leverage the United Way
The annual United Way campaigns are getting underway throughout the country. Last year many families had great success leveraging both the annual United Way campaign and Combined Federal Campaign (CFC) to raise money to fight Duchenne. Many people have a tradition of donating to the United Way, are expecting their company campaigns to start, and are looking for good causes to earmark their donations. Download PPMD's United Way/CFC Fundraising Campaigns guide (PDF) which provides information on how local United Way operate and how to work to arrange funding through them.
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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DuchenneConnect
Run For Our Sons
Coach To Cure MD
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© Parent Project Muscular Dystrophy 2015 — All Rights Reserved.

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