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Parent Project Muscular Dystrophy

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Go for the Gold
Go for the Gold
Dear Friend,

It’s Olympic Summer…that special time every four years where we all get chills hearing the fanfare, the lighting of the torch, watching our favorite events, and feeling connected to the rest of the world in a way that is unique to this sporting tradition.

For this community, though, the Olympics can be a painful reminder of how our sons are limited physically. In 2008, Olympic hopeful Ryan Lochte chose PPMD as his charitable cause because he had lost a family member to Duchenne. As he won medals and broke records, there was a personal connection not only to a great athlete, but someone who was a friend of this community and a champion - a champion for our sons.

For the 2012 Olympic Games starting next Friday, July 27 in London, Ryan is the face of the American team. His star is shining even brighter, with even more promise. He is in all the magazines, all the TV ads, being interviewed everywhere. And again, he’s swimming for us.

At PPMD’s Annual Connect Conference, we received a wonderful video message from Ryan. A message that challenges all of us to “Go for the Gold” and raise money for specific research projects so that we can be victorious too, and get that much closer to ending Duchenne.

To date, we have raised $189,120 of our $300,000 to fund SERCA2a and tamoxifen (learn more below). We hope that as you watch Ryan win more medals and break more world records in the coming weeks, you will join him in his support of PPMD by giving to our Go for the Gold campaign and fund this critical research that we believe will help all of our sons dream Olympic dreams, and one day see those dreams come true.

Tell your family and friends that your son has a pal in the Olympics this summer - an athlete and hero that believes your child deserves to dream the same dreams he has achieved. And ask them to support this campaign.

Together, let’s join Ryan and Go for the Gold!
Together, we will end Duchenne.

Let the Games begin!

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Pat Furlong
President & CEO
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SERCA2a gene therapy for Duchenne cardiomyopathy.
As gene therapy approaches for Duchenne advance slowly but surely, Dr. Dongsheng Duan of the University of Missouri is testing in mice a new gene therapy approach to treating the heart—in this case, instead of delivering only a healthy copy of a mini-dystrophin gene (made smaller to fit inside the viral carrier), Dr. Duan will try delivering the gene for a protein called SERCA2a that helps regulate calcium in the heart either alone, or in tandem with a mini-dystrophin gene. The concern is that the mini-dystrophin gene alone may not fully treat cardiomyopathy in Duchenne. The SERCA2a gene is already in clinical testing for general heart failure so this technology may move faster when applied to Duchenne than a gene therapy that has never been tested in humans before.
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Preclinical investigation of tamoxifen in mdx mice as prerequisite for a clinical trial in Duchenne patients.
Tamoxifen is the generic name for an approved drug that is used to treat estrogen-dependent breast cancer. In breast tissue the drug blocks estrogen receptors and slows the development of cancer. In other tissues the drug may stimulate estrogen receptors instead of blocking them. Recent data from Dr. Urs Ruegg’s laboratory at the University of Geneva has shown that tamoxifen can trigger near normal improvements in muscle strength in mice that lack dystrophin. Dr. Ruegg has proposed additional experiments to PPMD that will help resolve questions about the mechanism of action of this drug in mice that lack dystrophin, and will also determine how Tamoxifen interacts with prednisone when the two are given together. The answers to these questions are needed to plan a human clinical study of this drug in Duchenne.
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At PPMD’s Annual Connect Conference, we received a wonderful video message from Ryan. A message that challenges all of us to “Go for the Gold” and raise money for specific research projects so that we can be victorious too, and get that much closer to ending Duchenne.
Watch on Youtube
Donate Now
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
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© Parent Project Muscular Dystrophy 2012 — All Rights Reserved.

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Let's seize this moment.


There has never been so much momentum in the Duchenne community. Help us keep moving forward by donating to fund CRISPR/Cas9 gene‑editing technology. Every gift to PPMD is being doubled until we raise $250,000—but we have to reach this goal by 12/31!