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Landmark Meeting with the FDA
May 2012
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Just over a week ago, I had the privilege of meeting with leaders of the Food & Drug Administration's (FDA) drug review center to seek ways to accelerate the review process and to ensure the patient voice is included when key decisions are being made at the FDA (READ THE RELEASE). The importance of this meeting did not go unappreciated by me or the small handful of PPMD Board Members, parents, and experts that attended. We knew we spoke for the Duchenne community as we shared our stories and your stories, and stressed to the FDA the importance of time. Minutes matter. We walked away with the best news we could realistically hope for - more meetings in the future, a plan in place. Patience may be a virtue, but I'm sure many of you agree, when Duchenne has invaded your family, it is not a virtue easy to live by. April was a busy month, with an incredibly successful West Coast Connect Meeting in San Diego (download presentations now!) and a trip down into (and back out of - they don't tell you that part!) the Grand Canyon, as we took part in our first Conquer the Canyon event. We head into May, with plans underway for an amazing Annual Connect Conference in Ft. Lauderdale and an agenda that is like nothing we've done before. And we introduce you to a remarkable young person, Cory Stalling who is working with his parents to raise awareness by using social media and building a team of supportive runners!

The calendar is full...Full of projects, full of "to-dos", and most importantly, full of promise!
Pat Furlong, PPMD Founder & President

Cory Stalling, 11 years old
Research
AVI Presents Additional Data from Phase IIb Eteplirsen Study
On April 25, 2012, Dr. Jerry Mendell of Nationwide Children's Hospital presented additional data from the Phase IIb study of Eteplirsen, currently ongoing. Check out Dr. Mendell's presentation. This follows their April 2 release stating that Eteplirsen met primary endpoints. You can read PPMD's Director of Clinical Care, Kathi Kinnett's blog for more information.
DuchenneConnect Direct Access Webinar Series
Dr. Stanley Nelson, Professor of Human Genetics at the David Geffen School of Medicine at the University of California, Los Angeles, discusses his study of the DuchenneConnect data. His research focused on analyzing the registry data from the start of the registry in 2007 through June, 2011. Watch the webinar and find answers to questions asked during the presentation.
Help DuchenneConnect Help You
We are planning a Pre-Conference Clinical Meeting for healthcare professionals before PPMD's Annual Connect Conference in June. We are trying to optimize care in clinics across the country. In order to do that, we need to know what care looks like in your clinic. We want to know what is available in your clinic and how satisfied you are with the care in your clinic. Please take 20 minutes to complete this important survey. A response by the end of May will allow us to use your survey data for our Pre-Conference Clinical Meeting in June.
Other Research News
SMT C1100 approved to enter Phase I clinical trial. PPMD was an early funder of this research.
If you were unable to join us in person or via live streaming for the West Coast Connect Meeting, click here for presentations.
Advocacy
PPMD Holds Landmark Meeting with FDA Leaders
PPMD convened a high-impact meeting with leaders of the FDA drug review center to seek ways to accelerate the review process and to ensure the patient voice is included when key decisions are being made. The session represented a landmark meeting between high-level FDA leaders, and included the leader of the agency's Center for Drug Evaluation and Research, as well as, the head of its neurology products division. Read the press release to learn more about next steps.
Tell your Member of Congress to Endorse FAST today!
There is still time to get your Member of Congress to cosponsor critical legislation aimed at accelerating drug approval for rare disorders. PPMD officially endorsed HR. 4132, the Faster Access to Specialized Treatments (FAST) Act, bipartisan legislation introduced a few weeks ago in the House. It only takes a few clicks and a phone call. With the House scheduled to mark up the Prescription Drug User Fee Act legislation (PDUFA) next week, now is the time to weigh in on bills that will factor into the FDA user fee bill.
Expanding and Promoting Expertise in Review of Rare Treatments (EXPERRT) Act
Along with FAST, PPMD has endorsed the Expanding and Promoting Expertise in Review of Rare Treatments (EXPERRT) Act, aimed at fostering greater FDA engagement with patient organizations. PPMD sent a letter to the Senate HELP Committee in support of this measure and more recently coupled this action with a letter to the House Energy and Commerce Committee as they finalize PDUFA legislation. PPMD is in full support of enhancing FDA's engagement with external experts, to include the patient voice to weigh in on key decisions.
Community
One Step to End Duchenne
PPMD is honored to have Tim Rice, great uncle of Jonathan Rice, embarking on a 500 mile hike along the Pacific Crest Trail in honor of all patients living with Duchenne. Tim is taking 1 Step to End Duchenne, as he says, 'because I can.' Join us in supporting Tim on his journey any way you can and check out his website for blog and video updates from the trail. Thank you Tim!
Run For Our Sons
This month's runner spotlight is on Julie Toy, a teacher who is running her third full marathon (the Bank of America Chicago Marathon on October 7th) in honor of two boys in her community who are living with Duchenne. Meet Julie!
Upcoming Races
Rock 'n' Roll Seattle Marathon & Half Marathon, June 23
Disneyland Half Marathon & 5K, September 2*
Rock 'n' Roll Philadelphia Half Marathon, September 16*
* Still recruiting! Join the fun and go the distance to end Duchenne!
Conquer the Canyon
PPMD President Pat Furlong and 11 other hikers trekked through the Grand Canyon as part of our first team to Conquer the Canyon to End Duchenne. Together they raised nearly $40,000...Congratulations! Read Pat's blog recounting the experience, along with some great photos from the journey. And join us next year May 3-6, 2013!
Living with Duchenne - A Facebook Phenomenon!
We are so excited that our Facebook image "Living with Duchenne' has received such positive feedback and been shared by so many people. We think this piece explains Duchenne to that person on the street who may not get what living with Duchenne means to the people it affects. If you haven't already, share this image with your online friends! And make sure you check out PPMD's gallery of images you can use for the new timeline format. Visit our Facebook page to choose the perfect image for your profile.
Dine Out for Duchenne
It's almost time! PPMD is excited to announce Dine Out for Duchenne, a week-long, online event where people can purchase gift certificates donated by restaurants throughout the United States. These gift certificates will then be displayed and sold on our Dine Out for Duchenne website during the week of May 13-20, 2012. Please start spreading the news and help us get people excited about the launch of this new event!
My Voice: Cory Stalling, 11 years old
Cory Stalling is a fifth grader from Missoula, Montana. On July 8, a Run For Our Sons team will again pound the streets of Missoula on behalf of Cory as part of the Missoula Marathon. Cory and his family have been a big part of this community and now are taking another step to raise awareness, with a just launched video about Cory, Run For Our Sons, and Duchenne. Cory is a young man who is living his life, not letting Duchenne slow him down, grateful for the people who support him. His drive to always challenge himself is one of the reasons Cory is our featured voice for May. Meet Cory.
Upcoming Events
• May 6: Cincinnati Flying Pig Marathon
• May 6: Lincoln National Guard Marathon & Half Marathon
• May 13-20: Dine Out for Duchenne
• May 14-18: Spike Duchenne
• May 18-19: Omaha Beerfest
• May 19: Kids Dash for Micah's Miracle
• May 20: Rite-Aid Cleveland Marathon
• May 20: Swing Time
• May 20: Bay to Breakers 12K
PPMD is happy to introduce an updated Calendar section on our Community Site. Not only is it easy to find out information about all the great events going on in our community, you can also save it to your own personal online calendar! Check out the Calendar.
Education
Agenda for PPMD's Annual Connect Conference Available Now
PPMD is extremely proud to present the next meeting in our 2012 educational series, the Annual Connect Conference in Ft. Lauderdale, FL, June 28 - July 1, 2012. This year PPMD will be hosting a three part series on Clinical Trials. We will hear panel presentations from parents whose children are or were participating in trials, from clinicians who run the trials, and from industry leaders themselves who develop the trials. These interactive panels will give us a glimpse into the future of Duchenne trials and hopefully enlighten us all. And to make it a true family event, Kids Track will return for the third year, giving children of all ages an opportunity to feel engaged at the conference. Check out the agenda and register today!
If You Had a Chance...
To ask researchers, providers or industry any question at all about clinical trials, what would it be? In anticipation of the panel format at this year's Annual Connect Conference in Ft. Lauderdale, we wanted to reach out to you - the Duchenne community - and find out the questions you would ask if you had a chance to ask researchers, providers, or industry any question at all about clinical trials. Tell us your question and we will submit them to these expert panels!
Educational Resources Everyone Can Use
Jill Castle, a Parent Information Network Specialist for the Arizona Department of Education and the PPMD FACES of Arizona Volunteer Coordinator, has put together a collection of important educational tools for you to have at your fingertip, as you navigate the complicated and ever changing environment of your child's school and school system. Download the resources that pertain to your situation today.
Kitchen Table Conversations: Mothers & Daughters
In honor of Mother's Day, the latest HerSelf First podcast features special guests Kris Hersom from Florida and Christine Piacentino from Rochester, NY discussing the complexities of the mother-daughter relationship, including what their daughters are teaching them about taking risks, living fearlessly, and staying connected to their sense of self. Listen now.
Two Moms, Co-Blogging: Priorities
Getting it all done and figuring out what needs to be done first. Everyone, whether you are dealing with Duchenne or not, feels the pressure of time and faces a daunting, ever-growing to-do list. But when you're dealing with a disorder like Duchenne, that clock ticks louder. In their monthly co-blog, PPMD President Pat Furlong and Colorado FACES coordinator, Ivy Scherbarth try to sort through priorities and what in our lives deserves more of our time and energy. Please take a moment after you read this month's co-blog to share your insights about priorities.
More Education Updates
Now Recruiting: HerSelf First's Survey for Moms
Make it Happen!
2012 Connect Conference
Make sure you and your family are registered for the Annual Connect Conference in Ft. Lauderdale, FL, June 28 - July 1, 2012. There is strength in numbers and comfort in connecting with other families on the same journey as you. Plus it is your opportunity to interact, face to face, with the leaders in Duchenne research who are working so hard to find treatments that will help your child. Register you and your family today!
Double Your Donation
These days, many companies will match your donation to a nonprofit organization. Find out if your company matches using our easy search feature. We are so grateful for every donation we receive. Why not see if your gift can be doubled?
Shop For Our Sons
Spring is in the air. Shower those that you love with great gifts from Amazon and help fight Duchenne while you’re at it. Visit ShopForOurSons.org and click the Amazon link. Then shop away. It’s that easy! Share this site and encourage your friends to shop for our sons!
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 2012 — All Rights Reserved.

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