Parent Project Muscular Dystrophy Receives Grant from NINDS

Oct 13, 2011, 14:10 ET from Parent Project Muscular Dystrophy

Grant to Help Largest National Duchenne Organization Fund Research Projects

HACKENSACK, N.J., Oct. 13, 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 received a grant from the National Institutes of Neurological Disorders and Stroke (NINDS), part of the National Institutes for Health. This grant was awarded to fund PPMD's ongoing interest in maximizing the clinical trial experience for participating patients with Duchenne muscular dystrophy.

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PPMD will survey and interview the Duchenne community regarding the clinical trial experience. According to PPMD's Senior Director of Education and Outreach, Holly Peay, MS CGC, "We have a great advisory committee of patients with Duchenne, parents, clinicians, researchers, and industry.  Our goal is to analyze hopes and expectations in the clinical trial experience, comparing reasonable hopes and expectation versus unrealistic ones so we can make recommendations that improve the experience for the participating families. This information will help inform clinicians as they prepare to take their prospective therapies through the clinical trial process. We are so grateful to NINDS for supporting the work of Parent Project Muscular Dystrophy."

As part of Parent Project Muscular Dystrophy's comprehensive approach to fighting to end Duchenne, Ms. Peay explains the importance of funding not just promising research therapies, but also staying in touch with the community to make sure patients are receiving exceptional care and the needs of families are being met. She continues, "While we are all fighting to end Duchenne, it is important for families to live well, today. PPMD is committed to working for both new therapies for our sons and happier days for our families. Not only are we continually supporting promising research, but we have several programs – including this one funded by NINDS – that seek to help families live better lives, albeit ones affected by Duchenne.  Yes, we are calling on the community to fill out surveys and participate in interviews. But PPMD is not just interested in gathering information and publishing in a journal. In addition, we get information from our families, and then turn that information into plans, recommendations, and interventions to benefit the community."

Other ongoing programs being sponsored by PPMD and supervised by Ms. Peay, include a mothers' survey in conjunction with HerSelf First and a clinic survey in conjunction with DuchenneConnect.

HerSelf First was created by Sheila C. Moeschen, Ph.D., and launched in May with the aim to equip caregivers with new tools to facilitate their long-term wellbeing and aid in their personal investment. The survey will collect information from mothers of children with Duchenne and Becker and will assess their needs, strengths, and everyday challenges. The results from the study will then be used to develop new interventions to improve mothers' wellbeing.  The mothers will be surveyed over the course of five years in order to analyze if and how a mother's wellbeing changes over time. Learn more.

DuchenneConnect, launched in 2007, serves as a central hub linking the resources and needs of those living with Duchenne and the professional community, including clinicians, policymakers, industry professionals, and medical researchers. The newly launched Clinic Survey allows families the opportunities to see how other patients have rated their local clinic. Then families can fill out a survey themselves, to share their own clinic experience. This information will be compiled and shared with the clinics and the community to improve care where needed and establish accurate models for the best care. Learn more.

Visit Parent Project Muscular Dystrophy's care section on the web, to learn more about HerSelf First, DuchenneConnect, and other programs that benefit patients with Duchenne and their families.

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.  

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

SOURCE Parent Project Muscular Dystrophy