HACKENSACK, N.J., Sept. 22, 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), announced that it has awarded Tejvir S. Khurana, MD, PhD, Professor of Physiology at the University of Pennsylvania and Pennsylvania Muscle Institute an End Duchenne Grant for $98,000.
Dr. Khurana is a molecular geneticist / physiologist and highly accomplished scientist who discovered the utrophin protein (originally named Dystrophin-related protein). This bridge funding will allow Dr. Khurana the resources to continue work to upregulate utrophin and correct the underlying defect in mdx muscle with the objective of translation to the clinic.
Duchenne muscular dystrophy occurs when the dystrophin protein is missing or not functioning. Utrophin is a kind of molecular cousin to dystrophin that can compensate for the lack of dystrophin when it is present in larger than normal quantities.
The End Duchenne Grant Award Program was created by PPMD in partnership with the National Institutes of Health (NIH) in an effort to ensure continuation of promising Duchenne research and translation to human studies. The End Duchenne Grant Award Program is a bridge grant provided by PPMD to selected research projects that receive scores beyond the current funding paylines of the NIH Institutes and Centers supporting Duchenne research. To date, PPMD has awarded over $1 million through this program. All past End Duchenne Grant recipients, thus far, have gone on to receive NIH funding.
Explains Pat Furlong, Founding President and CEO, "With its research budget growing tighter each year, the NIH can fund only a fraction of the many promising applications it receives. Currently, only those grants which score in the top percentiles are able to be funded. We are thrilled that we have been able to step in, since the launch of the End Duchenne Grant Program in 2009, and provide the bridge funding needed to continue critical research like Dr. Khurana's."
The End Duchenne Grant Award Program represents the first Duchenne specific bridging program and the first ongoing bridge funding program to be presented in the rare disease category. As a collaborative effort with the NIH, the selection strategy behind this award is very different than any other private research investment in Duchenne, yet the goal of this award remains consistent with all of PPMD's research initiatives: to advance promising research which will impact this generation affected by Duchenne.
Dr. Khurana looks forward to continuing his work upregulating utrophin and thanks the organization for their financial support: "Parent Project Muscular Dystrophy and University of Pennsylvania have a long history together. Receiving this End Duchenne Grant is a great affirmation that the Duchenne community believes in the work we are doing here. We are hopeful that this therapy will change the progression of Duchenne and help extend the lifespan and quality of life for all patients living with the disorder."
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, 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.
|SOURCE Parent Project Muscular Dystrophy|
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