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Pushing the FDA to Accelerate Treatments
October 2013
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This week brings us two webinars from two different companies working on exon skipping. GSK and Prosensa will host a webinar on October 15 discussing the results of the drisapersen trial, as well as what the future holds for them. On October 17, Sarepta will provide the latest results with eteplirsen and discuss their new Let's Skip Ahead program. Two webinars in one week from industry... times are changing.

Myself and several members of the PPMD team attended the World Muscle Society Congress week before last (check out our updates if you haven't already). It was overwhelming to see how this meeting has grown over the years. Days and evenings were packed with presentations on research and care. We were honored to have Holly Peay's Clinical Trials Expectation project recognized at the meeting. She was then asked to present her poster and the reception was fantastic. Clinicians, researchers, industry are not only interested in the work we are doing to help expedite the trial process; they are coming to us as a resource and because they know we are their connection to you.

Last week, PPMD published the white paper, "Benefit-Risk Assessments in Rare Disorders: The Case for Therapeutic Development in Duchenne Muscular Dystrophy as the Prototype for New Approaches." It suggests ways the FDA might make decisions differently for rare diseases, and especially for Duchenne. PPMD continues to engage with the FDA and will aggressively partner with them for changes that allow the approval process to move faster, with more and earlier access to treatments.

Grassroots events are in full force this month too, with exciting new events launching and the return of community favorites. One of these is the 6th Annual Brookhaven Family Run in Matthews, NC in honor of Jake Pritchard, an incredible 9 year old and our Featured Voice this month. Meet Jake and his family – maybe it will inspire you to start a family run in your community!

Everything in today's world seems to be moving at an accelerated pace. But our fight to end Duchenne can't slow down. If anything, we need to increase momentum, increase efforts, and work together as a community.

Thank you for always being involved and rolling up your sleeves when we need you.


Pat Furlong
PPMD Founder & President


Research
Pat Furlong Named Top 50 Orphan Drug Thought Leader
Congratulations to our Founding President and CEO, Pat Furlong for being recognized as one of the top 50 thought-leaders in orphan drugs and rare disease by the World Orphan Drug Congress! "Pat is probably the most renowned Duchenne Muscular Dystrophy advocate globally. Her brave and successful efforts in founding and developing the Parent Project Muscular Dystrophy organization has exponentially increased studies and new therapies for the disease." Check out the rest of the Top 50.
Webinars This Week
GSK, Prosensa, and Sarepta Therapeutics will be hosting webinars this week regarding exon skipping and the progress/next steps for their ongoing research. Learn more about each:
Notes from 18th International World Muscle Society Congress
PPMD President & CEO Pat Furlong, Vice President of Research Sharon Hesterlee, Vice President of Education and Outreach Holly Peay, Vice President of Clinical Care Kathi Kinnett, and DuchenneConnect Coordinator/Genetic Counselor Ann Martin attended the World Muscle Society Congress in California. A wealth of information was shared and our team has begun to report back some of what they learned while at this meeting.
Tivorsan Receives Additional Funding
We're gratified to see that Tivorsan has received additional funding from a local investment group in Rhode Island! In 2012 you helped us commit $500,000 to Tivorsan in milestone-tied payments to further its biglycan therapy for Duchenne. The company is on track to meet its next milestone before the end of the year. Read about Tivorsan's latest funder.
Other Research News
Let's Skip Ahead: This new resource from Sarepta Therapeutics offers an exon mapping tool that allows visitors to explore the potential link between specific genetic mutations in Duchenne and exon skipping.
Prosensa answers questions related to their relationship with GSK and the compound drisapersen, as well as questions related to their ongoing development platform prior to webinar.
GSK has also responded to many of the questions submitted via PPMD and our partners at UPPMD regarding drisapersen.
Download Podcasts from PPMD's Connect Conference
PPMD Launches Duchenne Central App
Advocacy
Pat Furlong Receives Lifetime Achievement Award
On October 10, PPMD Founding President and CEO Pat Furlong received the Patient Advocacy Lifetime Achievement Award from Rare Disease Legislative Advocates at the Rare Voice Awards Gala in Washington. While humbled and grateful to receive the recognition, Pat said in her acceptance speech that the award reflects the efforts of the entire Duchenne community, working as a single voice, steadily for over a decade in Washington.
PPMD Shares White Paper with FDA
PPMD, together with our partners in Washington, FaegreBD, have laid out the differences between benefit/risk assessment in common and rare diseases in a new white paper: Benefit-Risk Assessments in Rare Disorders: The Case for Therapeutic Development in Duchenne Muscular Dystrophy as the Prototype for New Approaches. This white paper has been delivered to the FDA, urging them to change their decision-making process when it pertains to rare disease, especially Duchenne. Read more about our latest efforts and how you can help us push forward.
Save the Date!
PPMD's Annual Advocacy Conference
Date: February 23-25, 2014
Location: Marriott Wardman Park
Save the Date
Other Advocacy News
What does the government shut down mean for orphan drug research, development, and approval? Rare Disease Report covers the shutdown's impact on the NIH and FDA.
Because of changes in health information resulting from the Affordable Care Act, the National Organization for Rare Disorders has provide a State-by-State Insurance Information webpage helping you to determine changes that may affect you and your family.
Community
Coach To Cure MD VI Helps #TackleDuchenne
Donations and events are still coming in for this season's 6th Annual Coach To Cure MD. But if you watched a televised game or attended a college game on September 28, chances are you saw one of the 11,170 coaches from over 625 universities that wore a patch to raise awareness. There will continue to be Coach To Cure MD events throughout the college and high school football season and we are so appreciative of the families who got in the game this year!

We also want to welcome and thank our new corporate partner Northwestern Mutual. There support will not only help fund critical research, but it will raise awareness and encourage others to help us #TackleDuchenne!
Run For Our Sons
Walt Disney World Marathon Weekend - Registration Deadline is Approaching!
Time is running out to join us for Run For Our Sons' 10th year celebration at the Walt Disney World Marathon Weekend! Join us January 9-12, 2014 for a magical weekend honoring you, our runners, for 10 years of running, walking, and rolling to end Duchenne. Disney has added two new races – a 10K and the Dopey Challenge, in which runners complete the 5K, 10K, half marathon and full marathon in four consecutive days! We'll have parties, meet & greets with Disney characters, hotel discounts, and much more. Register today and make memories your family will cherish forever!
Runner in the Spotlight
This month's runner spotlight is on Shane Lauer, a 21 year old man living with Duchenne who recently participated in his fifth half Ironman triathlon! This is his incredible story.
We need YOU!
Now recruiting for:
Rock ‘n' Roll Denver Marathon & Half Marathon - October 20
Anthem Richmond Marathon - November 16
Naperville Marathon - November 10
Philadelphia Marathon - November 17
Walt Disney World Marathon Weekend - January 9-12, 2014
Chevron Houston Marathon & Aramco Half Marathon - January 19, 2014
Rock 'n' Roll New Orleans Marathon & Half Marathon - February 2, 2014
My Voice: Jake Pritchard, 9 years old
When a family hosts a fundraising event for Duchenne, we are thrilled and extremely grateful. When a family hosts the same event six years in a row, we are overwhelmed! This year the Pritchard family in Matthews, North Carolina will host the 6th Annual Brookhaven Family Run. Jake Pritchard is the star of the day though, as families, friends, and neighbors gather to run on behalf of Jake – and all people with Duchenne. Jake is a 9 year old who understands the importance of what his family is doing. He's also fearless and not about to let Duchenne slow him down. Meet our Featured Voice for October and his great family.
Upcoming Events
• October 12: Baltimore Running Festival
• October 12: Argyle Run For Our Sons 5K/1Mile Fun Run
• October 13: Bank of America Chicago Marathon
• October 13: End Duchenne Bike Tour
• October 20: Rock 'n' Roll Denver Marathon & Half Marathon
• October 26: Brookhaven Family Run
• October 26: Boo Run 5K Run/Walk
• October 27: 2013 Marine Corps Marathon
• November 3: ING NYC Marathon
• November 9: GR .1K
• November 9: Sam's Soldiers Fight to End Duchenne Fun Run
• November 10: Naperville Marathon & Half Marathon
• November 17: Philadelphia Marathon
• November 23: Gunnar Listerman Memorial 5K
• November 27: Hustle 4 Muscle
• December 6: Hope for the Holidays Dinner & Auction
Check out our full Calendar and sync it with your personal calendar!
Save the Date!
2014 Connect Conference in Chicago
Date: June 26-29, 2014
Location: Chicago Marriott Downtown Magnificent Mile
Save the Date
Education
Important Flu Vaccination Updates for 2013-14
Flu season is back and the vaccine is slightly different this year. Nasal spray flu vaccines are not recommended. Injected flu shots are recommended for patients with neuromuscular diseases and patients taking chronic steroids. Learn more.
Living Duchenne: On Being Diurnal
"In the morning, under the radiance and warmth of the sun, in the brightness and hope of day, Life is Worth Living, whatever its form." It's amazing what the time of day can do to your mental health. FACES Coordinator Ivy Scherbarth explores being diurnal in her latest blog. Check out Ivy's blog.
More Education Updates
Meeting with your child's teachers and key staff is one of the most important things you can do for your child. Check out a list of resources we've compiled for parents and teachers.
Salt, Steroids, and Duchenne
Major Changes in Physical Therapy Coverage for Individuals with Duchenne and Becker
Make it Possible!
DuchenneConnect
Do your part – register on DuchenneConnect today! You will learn about actively recruiting clinical trials and research studies and how to participate, and you will help to advance research and speed treatments for Duchenne. Already registered? Please remember to update your Profile Survey at least once a year. Researchers need up-to-date data for their studies and we need this data to know what trials and studies are right for you or your child. Update your Profile now! Questions? Email coordinator@duchenneconnect.org.
Shop For Our Sons
Need a last minute Halloween costume ASAP? Starting your holiday shopping early? In charge of Thanksgiving this year and need pots and pans? Amazon has everything you can imagine. When you shop Amazon, using our link, we raise money for research!

Visit ShopForOurSons.org and click the Amazon link. Then shop away. It's that easy! A portion of all of your purchases will go to PPMD. Happy Fall!
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
Parent Project Muscular Dystrophy
PPMD Community
DuchenneConnect
Run For Our Sons
Coach to Cure MD
CONNECT
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© Parent Project Muscular Dystrophy 2013 — All Rights Reserved.

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