Parent Project Muscular Dystrophy Builds on Surprise Finding that Common Cardiac Drugs May Improve Limb as Well as Cardiac Weakness

Sep 20, 2011, 13:00 ET from Parent Project Muscular Dystrophy

Largest National Duchenne Organization Continues to Support Cardiac Health with Three New Grants in Duchenne Research to Ohio State and Nationwide Children's Research Institute

HACKENSACK, N.J., Sept. 20, 2011 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD), the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy (Duchenne), has awarded three separate $50,000 exploratory awards to researchers at The Ohio State University and the Research Institute at Nationwide Children's Hospital as part of its ongoing cardiac initiative for Duchenne research.

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Jill Rafael-Fortney, Ph.D. and Paul Janssen, Ph.D. from The Ohio State University and Federica Montanaro, Ph.D. from Nationwide Children's Hospital each received $50,000 to continue their work in Duchenne research. These exploratory grants allow investigators the resources needed to gather enough data to jumpstart their projects.

Duchenne muscular dystrophy is associated with a heart condition called dilated cardiomyopathy.  Cardiomyopathy is a form of heart disease that enlarges and weakens the heart muscle, preventing it from pumping blood efficiently. It progresses rapidly and is life-threatening in many cases. Cardiomyopathy does not necessarily cause symptoms, but specific tests such as Echocardiogram and Cardiac MRI are able to identify changes in the heart muscle that can predict the onset, progression, and severity of the dilated cardiomyopathy. Heart issues affect all patients with Duchenne.

The PPMD funding to Dr. Rafael-Fortney builds on previous findings in the July issue of the journal Circulation. Dr. Rafael-Fortney had postulated that the drugs lisinopril and spironolactone, commonly used to treat heart failure in humans, may have anti-fibrotic properties in the heart if used very early in mice with a form of muscular dystrophy resembling Duchenne.  The investigators were surprised to discover that the mice not only showed improvement in their cardiac function, but also showed an improvement in skeletal muscle weakness.  

Dr. Rafael-Fortney spoke about the significance this grant will have on her team's work: "This funding from PPMD will allow our investigators to determine if the combination of lisinopril and spironolactone is required to improve cardiac function and skeletal muscle weakness, or if either drug alone is adequate.  This project lays the groundwork for a human clinical trial with one or both of these drugs and we are indebted to Pat Furlong and Parent Project Muscular Dystrophy for believing in the work we are doing."

Additional funding to Dr. Janssen at Ohio State University will investigate other ways to strengthen cardiac muscle directly, while the grant to Dr. Montanaro at Nationwide Children's Research Institute will explore how the minidystrophin gene, used for gene therapy in Duchenne, is able to bind to other proteins in the hearts of affected mice.

Pat Furlong, Founding President and CEO of Parent Project Muscular Dystrophy said, "Our organization is moving aggressively to take advantage of drugs that are already approved and may be able to buy time for these boys right now. The approach complements our commitment to developing entirely new therapies. Anytime we can nurture a potential therapy that may help our children live longer, healthier lives, we have an obligation to the Duchenne community to take that chance. Not supporting such innovative research may cost our sons precious time."

PPMD launched its cardiac initiative earlier this year, and to date has awarded over $1 million to cardiac-focused research, reaffirming the organization's commitment to support therapies that will benefit all patients with Duchenne.  PPMD works with leading pediatric cardiologists, researchers, thought leaders, and the entire community to address heart issues.

Ms. Furlong congratulated the grant recipients saying, "Each of these individuals is making a significant contribution to the fight to end Duchenne. The heart is a muscle too, so it is critical that cardiac health is considered when investigating therapeutics in Duchenne. Dr. Rafael-Fortney, Dr. Janssen, and Dr. Montanaro, as well as the incredible institutions they are affiliated with, represent some of the most promising new developments in the Duchenne arena. We are so grateful that they are devoting their energies to our fight."

Learn more about Parent Project Muscular Dystrophy's cardiac initiative, by visiting

About Parent Project Muscular Dystrophy
Duchenne is a fatal genetic disorder that slowly robs young men of their muscle strength. Parent Project Muscular Dystrophy's  mission is to end Duchenne.

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, D.C., 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 for more information or  to learn how you can support our efforts and help families affected by Duchenne.

SOURCE Parent Project Muscular Dystrophy