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Critical Research Initiatives in Duchenne Muscular Dystrophy

  • Published: 2010-06-10 : Author: Parent Project Muscular Dystrophy
  • Synopsis: Duchenne the most common form of childhood muscular dystrophy is a progressive and fatal muscle disorder affecting boys and young men.

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Parent Project Muscular Dystrophy Funds Two Critical Research Initiatives in Duchenne - Dr. Dongsheng Duan and Nicholas Dobes to Receive Grants

Patricia A. Furlong, Founding President and CEO of 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 today that PPMD will be funding two critical, promising research initiatives in Duchenne.

Dr. Dongsheng Duan, Associate Professor of Molecular Microbiology and Immunology at University of Missouri and Nicholas Dobes, a student at University of North Carolina - Chapel Hill will each receive grants to help them continue their work in Duchenne research.

Dr. Duan, receiving $116,000 from PPMD, is developing adeno-associated virus (AAV) mediated Duchenne cardiomyopathy gene therapy. The goal of this project is to test different versions or "serotypes" of the AAV viral carrier to see which will work the best in the heart. The project will investigate AAV6, AAV8 and AAV9 and variations of these three serotypes.

Mr. Dobes is the recipient of the first Weisman Fellowship from PPMD. This $50,000 grant is awarded to a research student that has chosen Duchenne-specific science as their chosen path of study. Mr. Dobes is using micro-pallet arrays to help identify healthy cells for stem cell research, as well as developing a process to keep these cells alive for future research. Inspired by one of his best friends who lives with Duchenne, Mr. Dobes decided to focus his doctoral degree on analytical chemistry and applied for, and won, the Weisman Fellowship.

Ms. Furlong is thrilled to be awarding two such gifted researchers with additional funding for their Duchenne-specific projects. "When we started PPMD 16 years ago as a small group of parents and grandparents trying to get treatment for our boys, our messages, our pleas, often fell on deaf ears. Today, we have established leaders in molecular microbiology, like Dongsheng, and rising stars in analytical chemistry, like Nicholas, devoting their talents and passion to ending Duchenne. Part of our organization's mission is to support critical research with funding when it most needs our help. To be able to give financial support to Dongsheng and Nicholas is a great pleasure because their science reflects the promise for treatment for all those living with Duchenne."

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.

Parent Project Muscular Dystrophy (PPMD) is a national not-for-profit organization founded in 1994 by parents of children with Duchenne and Becker muscular dystrophy. The organization's mission is to improve the treatment, quality of life and long-term outlook for all individuals affected by Duchenne muscular dystrophy through research, advocacy, education and compassion. PPMD is headquartered in Middletown, Ohio with offices in Fort Lee, New Jersey. For more information, visit www.parentprojectmd.org


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