To view this email online, go to http://www.parentprojectmd.org/site/PageNavigato/end_duchenne_enews_nov11     Grateful for Promising Research & Passionate Advocates – November 2011 PERSON of the MONTH Brian Levin, our November Person of the Month, has served on the PPMD Board, attended several conferences, participated in four New York City Marathons, and supported Project Catalyst. But in his latest entrepreneurial endeavor, Brian has taken Duchenne awareness to a whole new level: Perky Jerky - caffeinated beef jerky that you can buy at your favorite retailer. This year, Brian attempted the New York City Marathon outfitted in 40 pounds of jerky. This is devotion to a cause and determination to raise awareness at any cost. And this is just one of the reasons we chose Brian Levin as our November Person of the Month. Meet Brian. ShopForOurSons.org The holidays are almost here and now is the time to get some great deals on Amazon and raise money for the fight to end Duchenne while you shop!  Click on the Amazon.com link from the PPMD web page. Tell your family, tell your friends, tell your neighbors, tell your co-workers. All you have to do is: 1) Remember to go to the ShopForOur Sons.org. 2) Click the Amazon link. 3) Shop as usual and raise money for PPMD at the same time! Now's the time to go to ShopForOurSons.org! Make 'em Laugh Madhouse Tees is a great online store to get fun t-shirts while supporting the fight to end Duchenne. Jared Aronson, the designer of all Madhouse Tees, was diagnosed with Duchenne at four. Though confined to a wheelchair and on a ventilator, he continues to design and run Madhouse Tees. Jared uses this endeavor to educate the world about physical limitations by saying, “Just because you have a disability doesn’t mean you don’t have creativity and passion.” Check out these great designs and purchase t-shirts before 11/20/11 and portion of the sales will go to PPMD. Shop PPMD! PPMD has some great new items in our store. Give a gift that has a message and tells a story.  From cook books to jewelry to cozy sweats, we have something for that special someone in your life. Check out our secure, online store today! Double Your Gift! These days, many companies will match your donation to a nonprofit organization. Find out if your company matches by simply clicking here. If you're place of work matches, they should come up in the search along with the simple steps you have to complete to have your gift matched. We are so grateful for every donation we receive. Why not see if your gift can be doubled?    Stay Plugged In Facebook Twitter RSS  YouTube Flickr Biglycan: Continued Government Support & "Hot Space" Project to Watch The last couple of weeks have been exciting for Tivorsan Pharmaceuticals and its academic collaborators, Justin Fallon, PhD and his research team at Brown University, for their ongoing work with biglycan. On October 19, Tivorsan Pharmaceuticals was selected as one of 2011’s Windhover’s Top 10 Most Interesting “Hot Space” Projects to Watch (read the release). The recognition stemmed from biglycan’s potential to address an urgent medical need through a unique mechanism of action, the progress of its development, project leadership, and potential for indication expansion and multi-level partnering. On October 26, Dr. Fallon and his team received $1.33 million in funding for the third year of a $5 million multi-year Cooperative (U01) Award that Dr. Fallon and Brown received in 2009 (read the release). This award provides funding and support for the accelerated preclinical development of biglycan as a novel disease-modifying therapeutic for Duchenne. Dr. Fallon was the first recipient of PPMD's End Duchenne Grant when the program launched in 2008. This bridge grant allowed Dr. Fallon to resubmit to the NIH and then receive the UO1. PPMD has supported Justin Fallon and Tivorsan’s biglycan from the beginning and is hopeful that this potential therapy, which could benefit all patients with Duchenne, continues to move forward with this great momentum.  Learn more about biglycan. Report from World Muscle Society Meeting Two weeks ago the 6th International Congress of the World Muscle Society was held in Almancil, Portugal. Several representatives from PPMD attended and have compiled notes based on presentations from the major pharmaceutical companies focused on Duchenne. Read Pat Furlong's blog about ACE-031 and Sharon Hesterlee's synopsis of multiple presentations.  Latest Project Catalyst Update As you know, Project Catalyst is a first of its kind research collaboration between PPMD and PTC Therapeutics. The goal of Project Catalyst is to identify new treatments for all patients with Duchenne, regardless of the genetic mutation responsible for their disease. Periodically, PTC provides an update on the progress of Project Catalyst. Check out the latest update. Recruitment Updates We continually try to keep you abreast of clinical trials recruiting and/or trials that have completed their recruitment. Below are important updates: * GlaxoSmithKline updates for 114044, 114117, & 114118 * MRI & Biomarkers for Duchenne Study (Imaging DMD) Please remember that your best resource for clinical trials is DuchenneConnect linking our entire community — patients and families, medical/research professionals, and the medical/pharmaceutical industry, and bringing you the latest information about current treatments and clinical trial opportunities. And don't miss the DuchenneConnect Direct Access Webinar Series, most recently featuring a discussion about genetic testing.   PPMD Signs on to Critical Congressional Letter PPMD joined other advocacy organizations in signing a letter to Congress expressing concern over current FDA guidelines for Special Government Employee (SGE’s) serving on Advisory Committees. Currently a provision regarding conflicts of interest for those serving on these committees, specifically those with a financial interest in the disease area, often impedes the ability to choose the best qualified candidates. It is the group’s contention that the Federal Advisory Committee Act and the Ethics in Government Act of 1978, which require that potential conflicts of interest be disclosed, are adequate safeguards. For a rare disease, like Duchenne, it is imperative that the best possible minds in Duchenne are serving on these FDA Advisory Committees. They are the only people who can fully understand the complexities of the disease.  Read the letter. NORD Releases Study on Orphan Drug Approval The National Organization of Rare Disorders released a report in support of its efforts for the FDA to codify a flexible approach to the approval of orphan drugs. It examines the basis for agency approval of 135 non-cancer orphan drugs since the Orphan Drug Act was enacted in 1983. PPMD President Pat Furlong is a member of the NORD Board of Directors.  Read the report. Advocacy Matters: Why Your Voice is Needed With Our Voice As the year wraps up (where did 2011 go!) and we plan for 2012, we hope that you will make it a New Year's resolution of yours to take part in PPMD's 11th Annual Advocacy Conference in Washington, D.C. on February 26 to 28, 2012. We are only as strong as the voices that speak with us in Washington, D.C., every February. If you are new to this community, take a minute to watch this great video from Pat Furlong and John Killian on the importance of joining us in Washington. If you have been part of this community, recharge your advocacy batteries with this wonderfully eloquent piece that reminds you why our sons need each of us to advocate for them. Please share this video too, so that others can see the important work this community does year round to end Duchenne. And don't forget to save the date for PPMD's 11th Annual Advocacy Conference. Northern California FACES Launches! FACES has added a new location to its ever-growing list of local community support for families fighting Duchenne. On December 4, 2011, Northern California FACES will launch in Sacramento. This lunch meeting will include remarks from PPMD President Pat Furlong, PPMD's Director of Outreach and Advocacy, Ryan Fischer, and HerSelf First's Sheila Moeschen. And of course, there will be plenty of time for families to connect and get to know one another. We are so excited to welcome this FACES group, and look forward to future meetings. Get detailed information about the Northern California meeting. RUN FOR OUR SONS: News & Events Many of us had a taste of winter last week, but the cold air and snow didn't damper training for this winter's busy Run For Our Sons calendar! Many of you may already be looking to the warmth of Spring, which will be here before you know it. Check out the upcoming Run For Our Sons calendar for a variety of events to meet the needs of every level of runner: **Congratulations to our 34 runners at the ING New York City Marathon that surpassed their team fundraising goal and will bring in over $300,000! ** It’s a Disney miracle! Registration has been extended for the 2012 Walt Disney World Marathon Weekend until December 6th. Now you’ll have even more time to decide between all of the exciting race options available this year – from the Goofy Challenge to the Family Fun Run 5K, there’s something for everyone! Click here for more info! ** PPMD is excited to announce a brand new fundraising adventure – Conquer the Canyon to End Duchenne! This 3 night/4 day trip to the Grand Canyon, which includes a day-long hike into and out of the Canyon, is a once-in-a-lifetime experience. You’ll enjoy the Grand Canyon in a way that few people ever will. Visit our website for more information. ** Run For Our Sons is heading to The Big Easy in March 2012, for the Rock 'n' Roll New Orleans Marathon & Half Marathon! Now in its 3rd year, this race is growing fast, and we’re excited to be a part of it. Head south with us and enjoy a fun-filled weekend in New Orleans. More information is available on our website. **For the first time ever, Run For Our Sons is participating in the Family Fun Run 5K as part of the 2012 Walt Disney World Marathon Weekend, and that means kids are following in the footsteps of their parents – literally–when it comes to fundraising. This month’s fundraising tip is about easy and safe ways to help your kids raise money and meet their fundraising goals. Check out some easy and creative tips for the whole family!  We Need Moms! As part of the Parent Project Muscular Dystrophy’s Women’s Project, we are happy to announce the launch of the mothers’ survey. This survey collects information from mothers of children with Duchenne and Becker: your needs, your strengths, and your wellbeing. The results from this survey will help us develop new interventions to improve mothers’ wellbeing. Learn more and share your experiences.  PPMD Receives Grant from NINDS PPMD announced that it has received a grant from the National Institutes of Neurological Disorders and Stroke (NINDS), part of the National Institutes for Health. This grant was awarded to fund PPMD's ongoing interest in maximizing the clinical trial experience for participating patients with Duchenne muscular dystrophy.  PPMD will survey and interview the Duchenne community regarding the clinical trial experience. Read the release. Two Moms, Co-Blogging PPMD President Pat Furlong and Colorado FACES Coordinator Ivy Scherbarth are both mothers of boys with Duchenne, but at completely different points in their journey. For the last several months, Ivy and Pat have shared a co-blog, discussing topics that all of us in the community can relate to. We hope that after you read these entries, you'll post your own thoughts on the topic. It is a forum to share experiences and to tell your story. Not only can you learn from what others share, but it will reaffirm that you are not on this Duchenne journey alone. Read the latest co-blog on the "haves" and the "have nots." SAVE THE DATE There are so many opportunities for you to help raise awareness and learn more about Duchenne. Check out PPMD's extensive online calendar. November & December Events November 6, 2011 ING New York City Marathon November 23, 2011 Hustle for Muscle 5K December 2, 2011 Hope for the Holidays January 6-8, 2012 Walt Disney World Marathon Weekend Did you miss us last month? For an archive of previous newsletters, click here. 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 2011 — All Rights Reserved. 401 Hackensack Avenue, 9th Floor Hackensack, NJ 07601 Help us end Duchenne. Donate today. Forward to a friend. Was this e-mail forwarded to you? Subscribe today.