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.
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!
Pat Furlong
President & CEO
PPMD
ABOUT SERCA2a
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. • Donate Now
ABOUT TAMOXIFEN
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. • Donate Now
RYAN'S VIDEO MESSAGE
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.
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.
