view this email online
Parent Project Muscular Dystrophy Donate Now
Raising Our Voices
February 2014
Facebook Twitter RSS Feed Youtube

This winter has been brutal and thanks to our groundhog friend, it seems far from over. But that doesn't stop our determination to descend upon Washington, D.C., February 23-25, to tell our stories and make our voices heard. This year we are asking Members of Congress to reauthorize the MD-CARE Act. The reauthorization of this bill is of monumental importance to all the muscular dystrophies because it means that we are on the radar of our government, that we matter. We may be rare, but we are known.

And we can't do it without your help. We hope you will be able to join us in person in Washington. But if you can't, there are still plenty of ways you can help share our message from home.

February 28, 2014 is Rare Disease Day. Voices have already been heard in Washington earlier this month. Now is not the time to stop. Now is the time to speak louder. Call, email, tweet, and visit.

One simple action means huge rewards for our children.


Pat Furlong
PPMD Founder & President


Research
Sarepta Therapeutics Announces 120 Week Results
Finally some good news for this community! Sarepta's 120 week stability data on both the six minute walk test, as well as, pulmonary function is good news for the Duchenne community. We congratulate Sarepta for their continued commitment to people with Duchenne and we hope the FDA will consider the accelerated approval pathway. Read more about what this news means for the community and check out Sarepta's press release.
Download: PTC Therapeutics Webinar
On February 5, 2014, Dr. Robert Spiegel, Chief Medical Officer at PTC Therapeutics, presented an update about PTC's Phase 3 study of ataluren, now known as the Ataluren Confirmatory Trial in DMD (ACT DMD). Watch the webinar here.
Imaging DMD
Imaging DMD is a natural history study. The objective of the study is to validate the potential of noninvasive magnetic imaging (MRI) and spectroscopy (MRS) to monitor disease progression and to serve as an outcome measure in clinical trials. Although the study is focused on ambulatory boys, the team is also committed to develop outcome measures for boys who no longer walk. Pat Furlong serves on the advisory committee and shares her thoughts behind this technology. Learn more about Imaging DMD.
Using Kinect to Measure Upper Body Function
Recognizing the need to enhance trial participation for those who are non ambulatory, we've invested in the development of endpoints to measure upper body function. One project we're funding uses the Microsoft Kinect system to measure the "functional space" that a person can reach. Find out how this innovative project works.
Other Research News
Shocking Secrets Of FDA Clinical Trials Revealed
Register for DuchenneConnect and stay connected to the latest updates on clinical trial recruitment.
Visit PPMD's Duchenne Research News Section for the latest Duchenne Headlines
Advocacy
PPMD's 2014 Advocacy Conference:
Our Most Important Year Yet!
It's not too late to register for PPMD's Annual Advocacy Conference in Washington, D.C., February 23-25. Your voice is critical in Washington, and as we gather in our nation's capital asking Members of Congress to support the reauthorization of the MD-CARE Act, we hope you will do your part to raise awareness about this critical legislation.
Why is the MD-CARE Act So Important?
This is a pivotal moment in Duchenne's history and in this time of austerity measures and other cutbacks by Congress we MUST keep Duchenne on the front lines of agency priorities. The return on investment to date has been extraordinary – with years added to lifespan, over $250 million invested into Duchenne, and several therapies in late stage development.

This will be an action-packed three days that include advocacy training, briefings on Capitol Hill, and of course the all-important office visits to your Representatives and Senators. Learn more and regsiter today.
Sign Up for PPMD Action Alerts
You and your friends can participate from home while members of the community visit offices on Capitol Hill. This will help make our message and the urgency of the reauthorization of the MD-CARE Act that much more potent. Sign up for PPMD's Action Alerts.
How You Can Help PPMD's Advocacy Agenda
PPMD's advocacy strategy is multi-agency (FDA, NIH, CDC) and multi-layered. On February 11th , Pat Furlong, Ryan Fischer, and Dave Zook (FaegreBD) provided an overview of PPMD's overarching advocacy strategy, laying out our goals for 2014 and discussing how you can get involved at our Advocacy Conference in Washington, DC and/or advocate from home. Watch the webinar.
FDA Guidance Update: Representing the Duchenne Community
On Monday, February 24, members of the Steering Committee and working groups will provide progress to date and may request additional comments and opinion about particular topics. We will also include a Community Advisory Board made up of members of various foundations that will be given an opportunity to give feedback as the policy is being drafted. This will take place in conjunction with our Annual Advocacy Conference. Find out what this meeting will entail.
What Does FDA's 'Breakthrough' Pathway Mean for Neurology
PPMD's Vice President of Research, Sharon Hesterlee, PhD, participated in a great piece published by Neurology Today on the potential impact of the FDA's latest expedited review program – the breakthrough therapy designation – on the therapy pipeline for Duchenne, discussing both drisapersen and eteplirsen. Sharon makes it clear that this community will not accept the FDA's lack of flexibility. We don't have time.
Advocacy Conference: Register Now!
PPMD's Annual Advocacy Conference
Date: February 23-25, 2014
Location: Marriott Wardman Park
Register Now
Other Advocacy News
PPMD Board Member, Bob McDonald, shares his Duchenne journey with Department of Defense Duchenne Muscular Dystrophy Research Program
Duchenne Illustrates Potential Utility of Innovative Benefit-Risk Assessments in Regulatory Review
FDA Public Workshop Report – Our Message Is Having an Impact
Community
SAVE THE DATE: Coach To Cure MD Returns
September 27!
Coach To Cure MD marks seven years of raising awareness when we return on Saturday, September 27. Last year was a landmark year for this great awareness-raising program with over 625 universities participating and 11,170 coaches wearing our CTC patch. Later this Spring, we will be reaching out for help to get your favorite college or university signed up. Coach To Cure MD is one of the most fun ways you can raise awareness and money to help us #TackleDuchenne!
Feature Film Focuses on Duchenne
Coming soon to a theater near you! Different Drummers tells the incredible true story of two boys in the 1960s – one with Duchenne and one with ADHD – who form a unique and powerful friendship. It's truly rare to see a feature film that captures Duchenne. The movie will be coming soon to a theater near you, but until then read this fantastic story by ordering your copy on Amazon through ShopForOurSons.org! And like their Facebook page for updates on where you can see Different Drummers.
Run For Our Sons
The Happiest Race on Earth
The Disneyland Half Marathon Weekend is SOLD OUT, but Run For Our Sons is your ticket in! We have a limited number of entries available for this super fun race weekend, August 29th-31st that takes you through Disneyland and Disney's California Adventure. Our 5K and 10K entries are filled, but we have half marathon and Dumbo Double Dare entries still available, so grab yours today for this magical race experience.
New Additions to our Race Calendar!
We've added two brand new races to our 2014 race calendar – the Kaiser Permanente Colfax Marathon in Denver and the DICK's Sporting Goods Pittsburgh Marathon. Join us in May for these exciting races and challenge yourselves on a whole new course. View our full race calendar.
Bike to End Duchenne
Participate on our first ever Bike to End Duchenne team as part of the Million Dollar Bike Ride in Philadelphia on May 3rd, hosted by the Penn Medicine Center for Orphan Disease Research and Therapy. Choose from three race distances: 11 miles, 34 miles or 75 miles. All of the money raised will fund research to end Duchenne. Learn more and register.
Runner in the Spotlight
"Cade, like all the boys faced with this disease, is courageous, spirited, and strong, but lacks the ability to express this through sport. I am so grateful to be able to participate in something that not only raises awareness, but makes him proud of his aunt and feel supported by his family and friends." Meet Carrie Bruhl, who runs for her nephew Cade and has raised tens of thousands of dollars to support the fight to end Duchenne.
A Fruitful Winter Race Season
We kicked off 2014 with three action-packed race weekends at the Walt Disney World Marathon Weekend, the Chevron Houston Marathon, and the Rock 'n' Roll New Orleans Marathon. Check out our race recaps and catch up on the fun!
Now recruiting:
Cincinnati Flying Pig Marathon & Half Marathon - May 4, 2014
DICK's Sporting Goods Pittsburgh Marathon - May 4, 2014
Kaiser Permanente Colfax Marathon - May 18, 2014
Rite-Aid Cleveland Marathon & Half Marathon - May 18, 2014
Rock 'n' Roll Seattle Marathon & Half Marathon - June 21, 2014
Disneyland Half Marathon Weekend - August 29 – 31, 2014
Bank of America Chicago Marathon - October 12, 2014
TCS New York City Marathon - November 2, 2014
Simple Actions Have Lasting Impact
There are so many ways we advocate for our children in our day to day life. Whenever we speak on their behalf – at school, to physicians, in front of government officials – we are advocating, insisting that they are treated fairly and receive the best care and treatments possible. And we hope, especially after reading through this month's newsletter, you will realize the importance of advocating for the Duchenne community in our nation's capital.

This community has been led by some incredible advocates. Grandparents like Cathy Gaglianone, Anita Bullers, and Gail McVicker who call on their neighbors to reach out to Members of Congress with our urgent plea to support the MD-CARE Act. Parents like Tom Cosgrove and Christine Piacentino who travel with us to Washington every year to tell their story. Board members like Bob McDonald who serves as a reviewer on the Department of Defense's DMD Research Program. And creative voices like Dana Edwards who had her kids make a sign with their "ask" and then watched it become a viral sensation.

We need to make noise. We need everyone involved. It's a simple action with a simple ask. Help make a difference!
Upcoming Events
• February 15, 2014: UF Student Physical Therapy Association Golf Outing
• February 23-25, 2014: 2014 Annual Advocacy Conference
• March 16, 2014: Shamrock Shuffle 5K
• March 29, 2014: Hope4AJ 5K
• April 5, 2014: 2014 Unionville Run For Our Sons 5K
• April 11, 2014: Dancing with the Spartans
• April 12, 2014: Pins for Duchenne
• June 21, 2014: Rock 'n' Roll Seattle Marathon & Half Marathon
• June 26-29, 2014: 2014 Annual Connect Conference
Check out our full Calendar and sync it with your personal calendar!
Connect Conference: Save the Date!
2014 Connect Conference in Chicago
Date: June 26-29, 2014
Location: Chicago Marriott Downtown Magnificent Mile
Save the Date – Registration Opens Soon!
Education
Your Clinical Trial Experience
We're working with industry to change the way clinical trials are designed and conducted in the Duchenne community, and we need your input. What are some of your concerns regarding clinical trials? What would make trials better? Whether your child has been in a trial or not, industry needs to hear your voice. Please fill out one of these surveys so that we can share our community's clinical trial expectations. And find out why one mom, Joanna Johnson, believes this kind of data can help all of our children.
National Health Council Launches Patient-Centric Health Care Exchange Platform
The National Health Council (NHC), a nonprofit association of health and patient advocacy organizations, including PPMD, has launched a new web platform, Putting Patients First, designed to help people with chronic conditions, their families, and caregivers better understand the new Health Insurance Exchanges and find insurance coverage that meets their individual health care and budgetary needs. Learn more by visiting the Putting Patients First website.
Living Duchenne: Power
In her monthly blog, Living Duchenne, Colorado/Wyoming FACES Coordinator Ivy Scherbarth delves into the powerlessness of a Duchenne diagnosis. But she also talks about the strength and hope found in this amazing community and what can be accomplished by working together. Read Ivy's blog and share with us your own feelings.
More Education Updates
Avoiding the Emergency Room: How to Protect Your Child This Winter
Guide to Disability for Healthcare Insurance Marketplace Navigators Released
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.
Simple Actions
We need the Duchenne community's voice to be as STRONG as possible in Washington, DC later this month. Don't see YOUR state listed? We need your voice! Register to attend the 2014 Advocacy Conference.

(And if your state is already listed, please consider making the voice of the Duchenne community in your state even STRONGER by adding to the number of advocates attending!)
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
Facebook Twitter RSS Feed Youtube
© Parent Project Muscular Dystrophy 2014 — All Rights Reserved.

Help us end Duchenne. Donate today.
Forward to a friend. Was this e-mail forwarded to you? Subscribe today.

Unsubscribe from receiving email, or change your email preferences.


Unsubscribe from receiving email, or change your email preferences.