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A Rich Drug Development Pipeline
April 2013
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This is the time of year when seeds that were planted months ago, start to make their way to the top of the soil and eventually blossom into flowers decorating our landscapes. The same could be said for the Duchenne therapy landscape. The seeds of various innovative research ideas, planted months and months ago (if not years ago), are now slowly making their way to clinical trial – some already in trial, and navigating the various phases. You can smell the hope in the air. You can hear our prayers in the night.

PPMD’s Vice President of Research, Sharon Hesterlee, PhD, recently wrote a piece outlining the various research strategies that are in the Duchenne pipeline. I don’t think anyone can read a piece like Sharon’s and not feel like something is coming. The question of course is always…when? And, will it be soon enough for my child?

The other question, though, that you may not be asking – but you should take comfort in knowing that PPMD is asking is – will we be ready? An approved Duchenne therapy is not the end. There is a “complicated ecosystem,” as Sharon puts it, that must be considered. Sufficient and efficient care will play a vital role.

There are brilliant minds at work on behalf of our children. They are working fast and furious to make their therapies a reality. This month we celebrate one of the future minds in Duchenne research, perhaps. A tenth grader who has turned a science project assignment into a passion – Matt Scheivert is working on research that shows early promise. Meet this talented young future researcher.

It’s hard to find peace when you are living with Duchenne in your family. But know that this is an incredible time, being led by incredible people and we will end Duchenne.

With hope,


Pat Furlong
PPMD Founder & President

Research
Drug Development Prospects for Duchenne
As mentioned in Pat’s letter, PPMD’s Vice President of Research, Sharon Hesterlee, PhD, has provided a detailed update to the drug development pipeline in Duchenne. PPMD is proud of the contribution we have made to these ongoing research projects. Read Sharon’s blog.
PPMD Funds Investigator to Build New Muscle from Skin Cells to Treat Duchenne
PPMD has awarded University of Minnesota researcher Dr. Rita Perlingeiro a grant for $220,000 to develop a therapeutic approach for Duchenne using a new type of "adult-derived" stem cell called "induced pluripotent stem cells" (iPSCs). iPSCs can be created from non-controversial sources like skin, have the ability to multiply indefinitely, and can be coaxed to become many different kinds of tissue types. Learn more.
PPMD’s 2013 Connect Conference in Baltimore:
Better, Faster, Now
PPMD returns to Baltimore’s beautiful inner harbor for the 2013 Connect Conference focusing on Duchenne and what is being done/what we can do – better, faster, now. From presentations by the leaders in Duchenne research, to top industry officials and leaders in D.C., to critical care information and advice to better life for you and your family, this conference has everything. Most importantly, it is your chance to connect with the people who make up this amazing community. Join us in Baltimore for this life-changing event. Learn more and register.
Update to the Latest Exon Skipping Report from Guenter Scheuerbrandt, Ph.D.
Over the last several years, Dr. Guenter Scheuerbrandt, a German biochemist, has been providing periodical updates on the work being done in exon skipping, internationally. Read an update to Dr. Scheuerbrandt’s February report entitled: “A future Duchenne therapy for you will depend on your personal dystrophin mutation.”
Other Research News
Sarepta Therapeutics and University of Western Australia announce exclusive worldwide licensing agreement for exon-skipping program in Duchenne
GSK's Drisapersen reaches primary objective in study
Summit outlines clinical development plans for Utrophin modulator program for Duchenne
Alpha 7 Integrin shows promise for Duchenne gene therapy
Sarepta Therapeutics announces Eteplirsen demonstrates sustained benefit on walking test through 74 weeks in Phase IIb Study
Advocacy
MD-CARE Act 2013 & What You Can Do
We continue to make progress on the Reauthorization of the MD-CARE Act. Thanks to you, we have already collected a number of cosponsors for the legislation. It’s a strong start and we must continue to tell our story to Congress and explain why renewing this critical legislation is paramount to all those living with Duchenne and all those diagnosed tomorrow and beyond. Please continue to send the message to friends and family and ask them to take action. Read more updates about the Reauthorization of the MD-CARE Act, including which Members have signed on.
A Mother’s Quest to Save Her Son
By now, most of you have heard Jenn McNary’s story and most likely signed her petition. Jenn is a great advocate for this community and has been using her voice in Washington alongside PPMD to help affect the kind of change critical to getting treatments approved for Duchenne. This article from the Burlington Free Press tells Jenn’s story from the beginning. We are proud to be a part of her journey and think you’ll be able to relate to much of what she has been through and continues to go through. Read the article.
More Advocay Updates
On March 26, PPMD President Pat Furlong, along with other members of the Duchenne community met with the UK's Shadow Minister of Health, Andrew Burnham in a meeting at Parliament
Update on PPMD’s Risk/Benefit in Duchenne Therapies program
Community
Run For Our Sons
Walt Disney World Marathon Weekend – 10th Anniversary Celebration!
2014 marks the 10th year that Run For Our Sons has participated in the Walt Disney World Marathon Weekend. This is where our run program began in 1994, with 80 runners on a mission. Join us as we celebrate 10 amazing years, millions of dollars raised, and the thousands of runners – like you! – who’ve made it all happen. Disney has added some new races this year including a 10K and a Dopey Challenge – 5 races in 4 days totaling 48.6 miles! We hope you’ll join us for our most magical race weekend of the year!
Marine Corps Marathon
Run For Our Sons is proud to be participating for the first time in the historic Marine Corps Marathon in Washington, D.C. on October 27, 2013. With the starting line uniquely situated between the Pentagon and Arlington National Cemetery, this race honors those who are actively protecting our country and our fallen heroes. This race sold out in less than 2 ½ hours but Run For Our Sons is your ticket in!
ING New York City Marathon
We’re returning to the Big Apple for one of the nation’s largest and most popular marathons. After last year’s unprecedented cancellation of the race due to Superstorm Sandy, we’re looking forward to a bigger and better race than ever before! Join us in New York City on November 3rd.
Runner Spotlight
This month's runner spotlight is on Florida mom Jen Tullio, who is training for her fifth marathon in honor of her son Gray. Running is Jen’s outlet for dealing with her stress of having a child with Duchenne, while making a difference at the same time. Share Jen’s story.
We need YOU!
Now recruiting for:
Rite-Aid Cleveland Marathon - May 19
Rock 'n' Roll Seattle Marathon & Half Marathon - June 22
Disneyland Half Marathon, 10K & Family Fun Run 5K - August 31 & September 1
Medtronic Twin Cities Marathon - October 6
Under Armour Baltimore Running Festival - October 12
Bank of America Chicago Marathon - October 13
Rock ‘n’ Roll Denver Marathon & Half Marathon - October 20
Naperville Marathon - November 10
New Hampshire Governor Declares
“Duchenne Muscular Dystrophy Day”
After 8th grader Rebecca Gowing learned that her sister’s classmate and friend had Duchenne, she took action to help raise funds and spread awareness, creating Art for a Cure for DMD. What started as an idea for a fundraiser evolved into Governor Maggie Hassan declaring Friday, April 5, Duchenne Muscular Dystrophy Day in New Hampshire! Thank you Rebecca for your incredible event and for raising awareness. And thank you New Hampshire for recognizing the fight to end Duchenne!
My Voice: Matt Scheivert, 17 years old
As you know, each month we feature someone in the community with Duchenne, that has done something really special to raise awareness and help us in our fight. This month, we are featuring 17-year-old Matt Scheivert, who doesn’t have Duchenne, but has already decided that he wants to continue to work in Duchenne research and advocacy. He plans to attend PPMD’s Connect Conference this June in Baltimore, to further his education and meet some of his research heroes. His passion to help our cause, on behalf of the kids with Duchenne in his school, is awe inspiring. Meet this brilliant high school sophomore and our Featured Voice for April, Matt Scheivert.
Upcoming Events
• April 20: Unionville 5K
• April 27: Memphis Brewfest
• April 27: Dance for Duchenne featuring Downtown Horns
• April 26-27: 2013 West Coast Connect Meeting
• May 5: 2013 Cincinnati Flying Pig Marathon
• May 18: Kids Dash for Micah’s Miracle
• May 19: Rite-Aid Cleveland Marathon 2013
• May 28: Spike Duchenne
Check out our full Calendar and sync it with your personal calendar!
More Community Updates
Herriman City declares day in honor of Mitchell Jones
In the race to end Duchenne, the one thing we don’t have is time. That’s why we need you to be as active as you can in our community. What will you do?
Education
Duchenne: An Intergenerational Issue – Grandparents
Can Help!
A Duchenne diagnosis affects the entire family and grandparents can oftentimes feel as helpless and alone as the parents themselves. They see the progression of Duchenne in their beloved grandchild and they watch their own child suffer, not knowing how they can “fix” their little one. PPMD grandparents, Gail McVicker and Anita Bullers have hosted breakout sessions for the last two years at PPMD’s Annual Connect Conference. They will return to this year’s conference in Baltimore to lead a grandparents lunch session. Read what the experience has meant to them over the last couple of years and check out their presentations.
Register for the 2013 Annual Connect Conference
Summer is just around the corner and with that comes PPMD’s Annual Connect Conference. We are excited to be back in Baltimore, Maryland this year and really excited about this year’s program. In 2013, PPMD’s Annual Connect Conference will focus on Duchenne: Better, Faster, Now! Learn more about this year’s agenda and register today.
PPMD’s West Coast Connect Meeting
If you’re unable to attend PPMD’s West Coast Connect Meeting in San Diego at the end of the month, make sure you catch most the day’s presentations streaming live. Visit our website for more information on how you can connect from home!
PPMD’s Cardiac Initiative
PPMD has an ongoing commitment to cardiac care, because the heart is a muscle too. Heart issues don't just affect some people with Duchenne; they affect ALL people with Duchenne. For the past several years, PPMD has heavily invested in the pursuit of optimal care, interventions, and research pertaining to the management and prevention of cardiomyopathy. In 2011, PPMD funded close to $2 million toward toward developing a comprehensive Cardiac Initiative.

We have learned so much, but there is still a tremendous amount to do to understand and improve heart health in people with Duchenne. For this reason, PPMD is committing an additional $1 million to bring together experts to understand cardiac needs and opportunities; further our exploration of novel cardiac treatments; and understand more about device and transplant interventions. Learn more about this critical initiative.
Living Duchenne: Silence and Noise
PPMD guest blogger Ivy Scherbarth tried a social experiment in her home with findings that may surprise you. Read her latest essay to find out what happened when she went silent. Is this something you have tried or are interested in trying? Let us know! Read Ivy’s latest blog.
Defy Your DNA
Defy Your DNA by Dr. Stephen Shrewsbury illustrates how the genetic discoveries of the past two decades are giving rise to an entirely new system of personalized medicine that will identify and avert diseases before patients become ill, utilizing a class of potent and targeted “gene-patch” medicines. This is an informative and insightful read for anyone in the Duchenne community. Visit Dr. Shrewsbury’s website to learn more.
Seeking Stories from PAS users Who Have Experienced Disasters
The Center on Disability at the Public Health Institute along with the Center for Personal Assistance Services at UC San Francisco is collecting emergency and disaster experience stories from individuals with disabilities who use personal assistance services (PAS). We expect these experiences will lead to valuable lessons, techniques, strategies, and skills. Share your experiences.
Other Education Updates
If you missed our webinar with MD STARnet, click here to watch the presentation.
Help us spread the word about clinical care for Duchenne! Complete the Clinic Survey.
The 2nd Annual Wellstone Center Muscular Dystrophy Parent/Patient Conference will be held at April 20 at Nationwide Children's Hospital's Education Center
Save the Date: PPMD’s Connect Conference, June 27-30, 2013 in Baltimore
Make it Possible!
Connect to Clinical Trials
Do your part – register on DuchenneConnect today! You will learn about actively recruiting clinical trials and research studies and how to participate, and you will help to advance research and speed treatments for Duchenne. Already registered? Please remember to update your Profile Survey at least once a year. Researchers need up-to-date data for their studies and we need this data to know what trials and studies are right for you or your child. Update your Profile now! Questions? Email coordinator@duchenneconnect.org.
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
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DuchenneConnect
Run For Our Sons
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© Parent Project Muscular Dystrophy 2013 — All Rights Reserved.

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