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New Year, New Energy
January 2014
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I must start by saying thank you – thank you to everyone in the Duchenne community who gave so generously to our holiday appeal this year! We not only met, but exceeded our $200,000 goal to be used to build a Duchenne Superhighway. PPMD was thrilled to receive such overwhelming support for this important project.

If you weren't able to give financially during the holidays, there are still plenty of simple actions you can take during the new year to help us end Duchenne. Run For Our Sons, PPMD's endurance program, is one of those ways and celebrates 10 years in 2014.

Advocacy, fundraising, participating in surveys, raising awareness, Coach To Cure MD – these are just a small handful of some of the ways you can make a difference or encourage others to help support our fight.

Thank you for continuing to believe in the work PPMD is doing. May 2014 be an incredible year for you and your family – and may it be a landmark year in the fight to end Duchenne!

Pat Furlong
PPMD Founder & President

Over $200,000 Raised for Duchenne Superhighway
We're thrilled to announce that over $200,000 was raised for our new Duchenne Superhighway. With 7 public companies and more than 15 biotechs focused on finding treatments for Duchenne, we saw the need to make sure that potential therapies can be tested as efficiently and quickly as possible. The Duchenne Superhighway will help to make this testing process faster and simpler. Thank you to everyone who supported this great endeavor! Watch our video to learn more about the Duchenne Superhighway. You can also view a recording of our webinar discussing the necessity and goals of the project, with guests from Halo Therapeutics, Prosensa, PTC Therapeutics, and Sarepta Therapeutics.
PPMD Funds Kinnect Project
Recognizing the need to enhance trial participation for those with Duchenne who are nonambulatory, PPMD has invested in the development of endpoints to measure upper body function. Read here about a PPMD-funded project to use the Microsoft Kinnect system to measure the "functional space" that a person can reach.
Latest Updates on Four Major Therapies
Over the last few weeks, the community received updates from four major therapies. Click below to learn more about the progress of each therapy as well as what these updates may mean for you or your child: And make sure you follow PPMD's research news updates through! or by visiting the PPMD homepage.
Other Research News
Decode Duchenne: Genetic testing for patients with Duchenne or Becker
Let's Skip Ahead: Explore the potential link between specific genetic mutations in Duchenne and exon skipping
The latest updates on clinical trial recruitment: Register for DuchenneConnect and stay connected
The latest Duchenne Headlines: visit PPMD's Duchenne Research News Section
PPMD's 2014 Advocacy Conference:
Our Most Important Year Yet!
Today is the day 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 training for first time advocates, briefings on Capitol Hill, and of course the all-important office visits to your Representatives and Senators. Learn more and regsiter today.
FDA Public Workshop Report – Our Message Is Having an Impact
Last week, PPMD staff were invited by the FDA to their meeting on Complex Issues in Developing Drugs and Biological Products for Rare Diseases. PPMD's Holly Peay reports back from the meeting, noting that our information, our message, and our urgency is having an impact.
Duchenne Illustrates Potential Utility of Innovative Benefit-Risk Assessments in Regulatory Review
In a recent guest blog on PhRMA, PPMD's VP of Education and Outreach, Holly Peay and Tim Franson (one of our D.C. partners/experts) explain how new approaches are required to expedite the development of treatments for rare diseases like Duchenne. The blog highlights why your participation in PPMD's 2013 pilot study aimed at understanding the risk tolerance and treatment preferences of the patient population was so important. Read the article.
Advocacy Conference: Register Now!
PPMD's Annual Advocacy Conference
Date: February 23-25, 2014
Location: Marriott Wardman Park
Register Now
Other Advocacy News
For those of you unable to attend PPMD's landmark Duchenne Policy Forum and want a recap, please read an in-depth report from PPMD's Vice President of Research, Sharon Hesterlee, PhD
Help Update the MD Action Plan - Please check out Brian's recent blog and read about how you can help inform the update
Celebrating the Heroes That Fuel Our Fight!
We ended 2013 asking you to tell us about the special people in your life that inspire you, that give you the energy to wake up every day ready to fight Duchenne. And the result brought tears to our eyes! Check out our online photo album of the kids and adults that fuel us. Who is the incredible person in your life who has Duchenne and how have they made a difference in their community? It's not too late to add your own story!
Run For Our Sons
Superheroes Descend on Disney!
"We will fight for our sons, we will unite for our sons, and yes, we will run for our sons." With those words, Mike Gaglianone, our parent speaker at our team pasta dinner, kicked off an incredible weekend at the Walt Disney World Marathon Weekend. All together, 171 runners competed in the 5K, 10K, half marathon and full marathon races and raised more than $315,000! Read the race recap.
Run at a Mile Altitude...with Zero Attitude
Run For Our Sons is thrilled to add the Kaiser Permanente Colfax Marathon to our 2014 race calendar! Join us in Denver, Colorado on May 17th and 18th and choose from among a 5K, 10K, half marathon, full marathon, or the very popular full marathon relay. Run through Denver's iconic landmarks and then celebrate lakeside at Denver's City Park after the race. Register today and run for those who can't.
Run in Style
Check out our brand new Run For Our Sons jackets! Available in men's and women's sizes, these super-stylish, water-resistant, lightweight jackets will keep you dry and warm whether you're running on the course or cheering from the sidelines! Let everyone know you're part of a very special team by ordering one today.
Calling All Spirit Runners!
Not a runner, but still want to be part of the team? Join any of our race teams as a Spirit Runner and do your part to end Duchenne. Spirit Runners raise funds and receive all the same benefits as our runners, but don't participate in races. If you're a great cheerleader and a great fundraiser, become a spirit runner today.
Now recruiting:
Rock 'n' Roll New Orleans Marathon & Half Marathon - February 2, 2014
Cincinnati Flying Pig Marathon & Half 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
Simple Actions Have Lasting Impact
A family of 11... 9 kids (the youngest with Duchenne), 2 parents and ALL of them participated in last weekend's Walt Disney World Marathon. Bob McDonald, a PPMD Board Member, and each member of his family ran in at least one of the Disney races. Even his youngest son Mark, with Duchenne, participated via wheelchair. Everyone in the family took a little time and effort (okay, maybe a lot!) and did something to help end Duchenne. Sometimes the simplest actions have the greatest impact. Read more.
Upcoming Events
• January 17, 2014: Ekdor 2013 Golf Tournament
• January 19, 2014: Chevron Houston Marathon & Aramco Houston Half Marathon
• February 2, 2014: 2014 Rock 'n' Roll New Orleans Marathon & Half Marathon
• February 15, 2014: UF Student Physical Therapy Association Golf Outing
• February 23-25, 2014: 2014 Annual Advocacy Conference
• 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
Avoiding the Emergency Room
PPMD's Vice President of Clinical Care, Kathi Kinnett, outlines a few things you can do to help keep your child out of the emergency room this winter as well as important information if you do find yourself in an emergency situation. Read her latest blog.
Help Launch an Innovative Project in Care
David M. Steinhorn, MD is working on a project that utilizes telehealth (high-quality, televideo communication via home computers, iPads, smart phones, etc) for patients with Duchenne and their families to help overcome problems related to distance from the Duchenne clinic, access to school, connection with friends, etc. All we need to get this project started is your input! Help bring Duchenne care into your home.
College Course Offered in Duchenne
A course on Duchenne is being offered by the Ohio State University College of Medicine and Nationwide Children's Hospital and is open to the public via real-time streaming and podcasts. Each lecture will be given by a different Duchenne or muscle disease expert or advocate. PPMD President Pat Furlong will be a featured lecturer in April. Learn more and watch online.
Guide to Disability for Healthcare Insurance Marketplace Navigators Released
The American Association on Health and Disability (AAHD) has announced the publication of a technical assistance guide for the National Disability Navigator Resource Collaborative (NDNRC). The "Guide to Disability for Healthcare Insurance Marketplace Navigators" is designed to inform navigators and other enrollment specialists about special considerations people with disabilities face as they shop for healthcare coverage.
More Education Updates
PPMD's Vice President of Clinical Care, Kathi Kinnett, goes deep inside a case study for pediatric heart failure. Learn more from this important cardiac summit Kathi just attended.
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
Donations are important and we are always grateful for every penny donated to PPMD. But there are plenty of ways you can make an impact in the fight to end Duchenne, without spending a dime.
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
Parent Project Muscular Dystrophy
PPMD Community
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© Parent Project Muscular Dystrophy 2014 — All Rights Reserved.

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