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Time to Connect
June 2012
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It's the official start of summer. And for our community, it's that time of year when we come together with caregivers, scientists, and industry for PPMD's Annual Connect Conference. This year's agenda is quite astonishing, reflecting the vast number of therapies in the works, the updates to care, our increased presence in Congress, and a panel series that will dissect the clinical trial process from all points of view. We will also host a meeting prior to the Connect Conference, a roundtable meeting called Transforming Duchenne Care. We have gathered leadership from top medical institutions, neuromuscular experts, and patient representatives to discuss in an open forum the ideal configuration, features, services, and structure of state-of-the-art family centered care for patients with Duchenne. PPMD is proud of the research we continue to fund, and supporting potential therapies is one of our top priorities. But we also know the importance of improving care, educating the medical community, raising awareness, and maintaining federal support. Looking at this month's newsletter, I'm proud of what this community continues to achieve, working together to end Duchenne. I'm also proud of one young woman who has made her voice heard in Washington the last several years: Elizabeth Heller. Elizabeth is the rarest of the rare - a woman with Duchenne. And she's telling her story and using her voice so that people know that while Duchenne tends to predominantly affect men, woman can be affected. We are a community of fiercely passionate people and exceptions to any sort of norm. We are a community of people that will get what we want...we will end Duchenne.

I look forward to seeing many of you in Florida and hope the rest of you enjoy the official start of summer!

Pat Furlong, PPMD Founder & President

P.S. Many of you have emailed me, asking what I'm up to or if PPMD was represented at a certain meeting. Each month, I will try to bring you a brief recap of some of the places we've been and what we've been doing!


Elizabeth Heller, 25 years old
Research
Halo Therapeutics Raises $1.1 Million to Expedite Phase 2 Study of HT-100
Halo Therapeutics, LLC, announced that it has received financial support totaling $1.1 million from 12 nonprofit foundations serving the muscular dystrophy patient community. Halo will use the funds to expedite the phase 2 study of HT-100, its lead drug candidate for Duchenne. PPMD is proud to be one of the participating foundations. Read the release.
PPMD Study on Expectations for Clinical Trials
PPMD is conducting a study to understand how the Duchenne and Becker muscular dystrophy community (parents, individuals with Duchenne or Becker muscular dystrophy, clinician researchers, translational researchers, and industry professionals) think and feel about potential therapeutics and clinical trials. The project will focus on motivations, hopes, expectations, and responsibilities. We hope you will add your thoughts and experience to this important study.
The Arts of Neuroscientists: Stanley Froehner
Some scientists find that their art and research build upon each other, but for Stanley C. Froehner, Ph.D. (PPMD Scientific Advisory Committee member), art and neuroscience are complementary but separate endeavors. In the lab, Dr. Froehner works to understand the dystrophin associated protein complex and its role in muscular dystrophies. Outside the lab, he uses photography to take a mental hiatus from his work. Read this great piece about a great friend and champion of this community.
2012 Charles C. Shepard Science Awards
Congratulations to Pat Furlong and her co-authors for their nomination from the CDC and Prevention and the Agency for Toxic Substances and Disease Registry (CDC/ATSDR) for the 2012 Charles C. Shepard Science Awards. Pat helped author the nominated article: "Quality Assurance for Duchenne and Becker Muscular Dystrophy Genetic Testing: Development of a Genomic DNA Reference Material Panel" published in The Journal of Molecular Diagnostics.
DuchenneConnect Updates: Studies Recruiting
New Cardiomyopathy Study for Duchenne Recruiting in Ohio
PPMD is funding a study taking place at two sites in Ohio: The Ohio State University (OSU) in Columbus and Cincinnati Children's Hospital Medical Center (CCHMC). Three visits to the study site will be necessary, at baseline, 6 months and 12 months. Interim blood draws at 1, 3, and 9 months can be done at any convenient lab. There is funding to help cover travel costs. Approximately 40 males with Duchenne will be enrolled in this study. Learn more.

All Sites Now Recruiting for GSK Exon 51 Skipping Trial
The purpose of this study is to determine if GSK2402968 is effective in the treatment of ambulant boys with Duchenne resulting from a mutation thought to be corrected by exon 51 skipping. Two doses of GSK2402968 and placebo will be used in this study. This study is sponsored by GlaxoSmithKline (GSK). Learn more.
Other Research News
Summit achieves SMT C1100 Phase 1 dosing milestone - PPMD is proud to fund this trial.
Scientists Discover Clues to Muscle Stem Cell Functions
Bradykinin restores left ventricular function, sarcomeric protein phosphorylation and e/nNOs levels in dogs with Duchenne muscular dystrophy cardiomyopathy
Advocacy
PPMD Leads Critical Letter for Rare Disease Community
PPMD has teamed up with EveryLife Foundation for Rare Diseases and Genetic Alliance to galvanize the rare disease community and send a strong message to Congress to ensure the most important provisions for the rare disease community were included in the final Prescription Drug User Fee Act (PDUFA) legislation. The provisions are aimed at accelerating Food and Drug Administration (FDA) review of therapies to treat rare diseases and to enable FDA to more aggressively engage external experts in reviewing candidate therapies. With Congress hoping to vote on the final measure before the July 4th recess, 119 rare disease organizations united together in an urgent letter to the key committees tasked with reconciling the legislation in the House and Senate.

PPMD will continue to keep the community updated as we move toward the final vote of the bill.

Read the press release
Learn more about what this legislation means for your child
Community
Save the Date: End Duchenne Gala
PPMD will host its first End Duchenne Gala on:
Thursday, December 6, 2012
7:00 pm - 12:00 am
The Metropolitan Club • New York, NY

Mark your calendars and plan to join us for an incredible evening that will bring together those determined to Change What's Next in the fight to End Duchenne. Visit EndDuchenneGala.org for more information.
One Step to End Duchenne
Congratulations to Tim Rice who has surpassed his original hiking goal of 500 miles and is well over 700 miles on the Pacific Crest Trail! Tim, who's great nephew has Duchenne, now plans to walk the whole length of the West Coast for our sons. This is a phenomenal man on an extraordinary journey telling our story. Follow his progress and support Tim as he supports us! Read his blog and visit his website.
Run For Our Sons
Our 2012 Chevron Houston Marathon team was awarded as the highest fundraising charity without a training program! Our incredible team of runners raised more than $210,000. Run For Our Sons was honored by the Houston Marathon Committee at a luncheon this spring. PPMD Board member Heinrich Meerman and his son Grant, who has Duchenne, created this video to accept the award.
Upcoming Races
Disneyland Half Marathon & 5K - the original & magical Disneyland running experience!
Bank of America Chicago Marathon - Chicago
Rock 'n' Roll Philadelphia Half Marathon - Philadelphia
2012 Rock 'n' Roll Denver Marathon & Half Marathon - Denver
2012 Baltimore Running Festival - Baltimore
2012 Grand Rapids Marathon - Grand Rapids
ING New York City Marathon - NYC
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! Watch this great video recounting the experience and introducing you to our fantastic team!
A Very Special Thank You
On June 1, Steve Desmond, co-author of A Life With A Purpose, with his son Francis Desmond, donated $5,000 to PPMD as a result of sales of this inspiring and beautiful book. Learn more about this great father-son endeavor and purchase a copy for yourself!
Dine Out for Duchenne: Wrap Up
For just over a week last month, we brought you Dine Out for Duchenne - a new fundraising event where parents obtained gift cards to their favorite restaurants for us to sell online at face value. The result was over $3,300 made and 101 certificates sold. We are excited by this first outing with Dine Out for Duchenne and hope you will help out when we use this same fundraising method for Coach To Cure MD in September! Special thanks to Judy Schneider and Alicia Malatesta.
My Voice: Elizabeth Heller, 25 years old
Elizabeth Heller, our Featured Voice this month, is a woman living a full life - student, activist, wife - and does it all, living with Duchenne. Considered to be a disorder that predominantly affects males, Elizabeth is an exception. Each year, Elizabeth joins PPMD in Washington to tell her story and to speak on behalf of those of us not there to speak for ourselves. She wants to pursue public office one day so that she can continue to give a voice to not only rare diseases but to those who are perhaps the rarest of the rare disease community. This is an amazing woman, changing hearts and minds with everyone she meets. We're lucky to have her as such a vocal part of this community and we celebrate her as our June voice. Meet Elizabeth Heller.
Upcoming Events
• June 23: Rock 'n' Roll Seattle Marathon & Half Marathon
• June 28-July 1: 2012 Annual CONNECT Conference
• July 7-8: Kick for a Cure
• July 7: 7th Annual Shootout for a Cure
• July 7: 2nd Annual Bill Smith Memorial 5K Run-Walk-Ride
• July 8: New York City Triathlon
• July 8: Missoula Marathon
• July 27-28: JOA Picnic in the Park (PDF)
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.
More Community Updates
FACES continues to grow with the launch of a new group in Ohio, led by Tammy Henegar on June 25 at 6pm. Pat Furlong will be attending.
Free park passes from the U.S. National Park Service for people with disabilities.
Education
PPMD to Host 'Transforming Duchenne Care' Meeting
In the days leading up to the Annual Connect Conference, PPMD will host the first Transforming Duchenne Care workshop. As mentioned above, the goal of this meeting is to bring together leadership from top medical institutions, neuromuscular experts and thought leaders, and patient representatives to discuss in an open forum the ideal configuration, features, services, and structure of state-of-the-art family centered care for patients with Duchenne. At the end of the workshop, the group will generate a consensus list of suggestions and recommendations that can be used to define and develop a model of care for patients with Duchenne. Ultimately, we intend to utilize the model to create a network of Duchenne Centers of Excellence. We will share the notes from this meeting with the community in the weeks following the conference. We are excited to bring together this group of decision makers to discuss the care of our children.
Streaming PPMD's Annual Connect Conference
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. We know that it is not possible for everyone interested in the conference to attend, for a variety of different reasons. That is why, for the last several years, we have streamed as much of our Connect Conference as possible. Check out the PPMD Connect Conference landing page the week of the meeting to see what sessions will be streamed and when. In the meantime, check out this year's robust agenda (featuring a panel series on clinical trials) so that you know what you want to watch from your computer. Please note, not all presentations will be streamed due to restrictions placed on us by the presenters. As always, we will post presentations within the first few weeks after the conference is over. Check out the agenda.
Duchenne Documentary Wins Award
The documentary "Duchenne with a Future: The Power to Live" recently won First Runner-Up at the annual American Academy of Neurology conference in New Orleans. Chosen from over 300 submitted films, the 5-minute-version of the documentary was awarded during the yearly conference, organized by AAN (American Academy of Neurology). The documentary was created by Jos Hendriksen (Clinical Neuropsychologist) and Debby Schrans (Psychologist), in a joint effort with director Jelle de Jonge. The movie was made possible with funding from Parent Project Muscular Dystrophy and Duchenne Parent Project Nederland (NL). Read a recent interview with the filmmakers and watch the 5-minute version of the documentary.
Kitchen Table Conversations: Inner & Outer Beauty
Download this provocative discussion on the impacts of beauty culture on women who are both caregivers and moms, presented by HerSelf First. This podcast also highlights the work of fashion photographer turned social entrepreneur Rick Guidotti, Director of Positive Exposure. Rick is the featured keynote speaker at the 2012 Connect Conference.
Two Moms, Co-Blogging: Measuring Up
We all feel like we are being held up to some predetermined standard - whether it is at our job, in school, or just walking down the street. In Duchenne, we compare our sons to other people's sons, to others with Duchenne. But who is to say what is right or normal? In their monthly co-blog, PPMD President Pat Furlong and Colorado FACES coordinator, Ivy Scherbarth acknowledge that measuring and comparing has its place, but what is really important is being your unique self. Read this month's co-blog and share your insights about priorities.
Make it Happen!
Etsy Angels' Charity Auction
Every now and then, someone steps up and supports this community that we barely know. Lori Ware and her Steps for Seph team at Walt Disney World this January, have a runner that just joined and wanted to do good. As a result, Etsy Angels have chosen Duchenne as the cause they will support for June. Check out their site and support this wonderful group that is supporting us! Visit Etsy Angels.
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
The kids are home for the summer but it doesn’t mean that learning should stop! Stock up on great books while they are home and do it for a great cause. 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
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© Parent Project Muscular Dystrophy 2012 — All Rights Reserved.

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