Travel Grant Funded For REVERSE-DBMD Clinical Trial

Online, November 14, 2011 (Newswire.com) - Travel Grant funded for REVERSE-DBMD Clinical Trial

The Ryan's Quest Foundation, a non-profit organization specific to Duchenne muscular dystrophy, announced today the award of their most recent contribution toward the fight to end Duchenne muscular dystrophy. The Foundation has awarded Kathryn Wagner, MD, PhD, of the Kennedy Krieger Institute, Johns Hopkins School of Medicine, Baltimore, Maryland a $76,500 grant that will ease the financial burden of families who are participating in the REVERSE-DBMD (Revatio for heart disease in Duchenne and Becker Muscular Dystrophies) trial. The grant was facilitated through a newly formed partnership of Duchenne Foundations, the Duchenne Alliance.

Dr. Wagner is the Director of the Center for Genetic Muscle Disorders at the Kennedy Krieger Institute in Baltimore, Maryland. The trial is a randomized, double-blind, placebo-controlled, phase II study testing the safety and efficacy of Sildenafil (Revatio). The goal of the trial is to determine whether a 6 month trial of oral Sildenafil compared to placebo improves cardiac function in Duchenne muscular dystrophy.

Duchenne muscular dystrophy affects skeletal, diaphragm and eventually cardiac muscle leading to paralysis, respiratory complications, and heart failure. The typical life expectancy ranges from the late teens to the mid-20s. Recent advances in medicine are extending the lives of those affected, and preservation of cardiac health is increasingly important as the boys age.

PDE5 inhibitors, like Sildenafil, are known to have cardioprotective effects in some models of cardiac damage. Researchers have recently shown that Sildenafil can prevent and reverse the pathological cardiac dysfunction in a mouse model of DMD. Sildenafil is FDA-approved for the treatment of erectile dysfunction and pulmonary hypertension. Two recent reports have described Sildenafil as well tolerated when treating non-Duchenne children and neonates diagnosed with pulmonary hypertension. These characteristics provide a strong rationale for a clinical trial of Sildenafil in Duchenne.

Ryan's Quest co-founder, David Schultz explains, "We are cautiously optimistic that trials such as Dr. Wagner's (testing of FDA-approved compounds) will provide a promising treatment in Duchenne muscular dystrophy in the near future. By providing a travel grant, we hope to facilitate recruitment of participants and fill the trial to its capacity, thereby providing reliable and valid outcomes."

Further information concerning this ongoing trial can be found at http://clinicaltrials.gov/ct2/show/NCT01168908

"We are tremendously grateful for the generous support provided by Ryan's Quest for families who are traveling to participate in the REVERSE-DBMD trial. Traveling to participate in a study like this is often difficult, and it can be even more challenging for people with DBMD. We are thrilled about the potential for this trial to improve treatment of cardiomyopathy in DBMD, and this donation will really help people to participate." Daniel P. Judge, MD, Co-principle investigator, Associate Professor of Medicine, John Hopkins University.

The Ryan's Quest Foundation was founded by Hamilton, New Jersey residents David and Maria Schultz on behalf of their son Ryan and thousands of other young men diagnosed with Duchenne muscular dystrophy, a life limiting disorder for which there is no cure. Ryan's Quest is dedicated to finding a treatment that will ensure the survival and quality of life for this generation of affected young men and boys. The travel grant was proposed by Dr. Wagner's team through a software provided to the Duchenne Alliance members - the DuchenneDashboard. "We were very excited to utilize the Dashboard as a way to expedite our funding to Dr. Wagner, time is not a commodity in Duchenne". For more information on The Ryan's Quest Foundation, please visit www.ryansquest.org. For more information on the Duchenne Alliance, please visit www.duchennealliance.org.

The Duchenne Alliance was co-founded by The Ryan's Quest Foundation and several other Duchenne Foundations in an effort to provide continuation of promising Duchenne research. The DuchenneDashboard (www.duchennedashboard.org) is a web-based conduit to expedite the workflow of Duchenne Foundations and researchers, allowing greater communication and the ability to share resources in a secure environment.

Duchenne muscular dystrophy occurs in approximately 1 out of every 3,600 male births. It is considered one of the most common and severe forms of muscular dystrophy. Considered a genetic disorder, approximately 35% of all cases appear to be by spontaneous mutation. Duchenne is typically diagnosed between 3 and 7 years of age, with the mean year of diagnosis around the age of 5. Duchenne follows a predictable progressive clinical course of muscle weakness. Loss of ambulation occurs by the age of 12 and death generally in early adulthood secondary to respiratory or cardiac failure.

Contact: David Schultz
609-947-3611
[email protected]

One hundred percent of funds raised through The Ryan's Quest Foundation are directed towards research for a treatment and cure of Duchenne muscular dystrophy. To support the efforts of The Ryan's Quest Foundation, please visit www.ryansquest.org.