Ryan Lochte - End Duchenne: Ryan Lochte

RYAN'S GOAL

MEET ANOTHER RYAN

Ryan Karlin is 10 years old and living with Duchenne. His dream is to be a member of the USA Olympic Hockey team or a sports announcer so he can make games fun for the fans.

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Ryan Lochte - End Duchenne

Olympic Champion Ryan Lochte lost a member of his family to Duchenne muscular dystrophy, a fatal disease that robs boys of their muscle movement. Ryan is dedicated to the fight to end Duchenne and hopes you will join him.

How can I join Ryan Lochte to end Duchenne?

Ryan is challenging us all 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.

Please help us reach our goal of raising $300,000 to move forward therapies that have the potential to help all boys with Duchenne.

Join Ryan Lochte to end Duchenne

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What research projects will my donation fund?

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.

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.