Dr. Duan and his team at the University of Missouri want to determine if delivering the gene for the calcium-handling protein SERCA2a is sufficient to correct cardiac disease in mdx mice or if the dystrophin gene must be delivered as well. This is important information since SERCA2a gene therapy is in human testing now for non-Duchenne related cardiomyopathy.
Heart failure is a leading cause of death in people with advanced Duchenne. Through the course of the disease, heart function is progressively compromised by calcium overload and inadequate transport, which leads to cell imbalance, dysfunction, and death. SERCA2a is a calcium ion pump that may be able to reduce the damage caused by calcium overload.
PPMD President and CEO Pat Furlong stressed the importance of cardiac research like Dr. Duan's and his team: "The heart is a muscle too. This is something we stress over and over again in our fight to end Duchenne. Talented researchers like Dr. Duan understand that the best therapies in the world will do patients no good if their hearts can't handle the therapy. Therefore, this research will give us the building blocks we need to eventually create wholly successful therapeutics that patients can withstand. We are so thankful that Dr. Duan and the University of Missouri are part of this community!"
PPMD has been a long supporter of Dr. Duan's work, awarding him an End Duchenne Grant in 2010 for Duchenne-specific cardiac research.
Dr. Duan thanked PPMD for continuing to fund his work. "PPMD has made significant contributions to the development of effective therapeutic strategies in Duchenne muscular dystrophy. They understand that no single therapy or initiative is going to end Duchenne, but that it will take a broad approach. Our research will be critical in maintaining optimal care in patients so that treatments can provide the maximum effect. I appreciate the faith PPMD has had in my work over these last years, and will continue to explore cardiac issues in Duchenne."
Funding for this grant was provided by the Duchenne community and the PPMD Board, who participated in a 2012 "Go for the Gold Campaign," celebrating the efforts of Olympic champion Ryan Lochte, a longtime supporter of Parent Project Muscular Dystrophy. Ryan lost a family member to Duchenne and has joined the PPMD team to raise awareness and resources.
About Duchenne muscular dystrophy Duchenne, the most common form of childhood muscular dystrophy, is a progressive and fatal muscle disorder affecting boys and young men that causes the loss of muscle function, wheelchair dependency and a decline in respiratory and cardiac function.
We invest deeply in treatments for this generation of young men affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite, and educate the global Duchenne community.
Everything we do—and everything we have done since our founding in 1994—helps boys with Duchenne live longer, stronger lives. We will not rest until every young man has a treatment to end Duchenne. Go to www.ParentProjectMD.org for more information or to learn how you can support our efforts and help families affected by Duchenne.