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The Parent Project for Muscular Dystrophy Research Inc

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Nonprofit Overview

Causes: Health, Nerve, Muscle & Bone Diseases Research

Mission: Parent Project Muscular Dystrophy's mission is to improve the treatment, quality of life and long-term outlook for all individuals affected by Duchenne muscular dystrophy (DMD) through research, advocacy, education and compassion.

Programs: Support for research to accelerate treatments and a cure for duchenne muscular dystrophy including studies in the following areas: fda draft policy guidance, combined therapeutics, serca2 gene therapy, aon-induced exon skipping ips cells and therapeutic applications, and failed regeneration in md, to name a few.

educational initiatives including online resources an annual conference held in chicago that brought scientists and families in the duchenne community together to discuss and expedite treatments, patient care, and research for a cure for the disease. Other educational efforts were promoted through a new initiative called transforming duchenne care, f. A. C. E. S. , decode duchenne, and duchnne connect.

advocacy is promoted on an annual basis through a legislative conference held in washington, d. C. Under the leadership of a consulting firm based in the area. Parents take a hands-on role in communicating with legislators during this process, for the purpose of promoting awareness and encouraging government funding for research and patient care.

Community Stories

2 Stories from Volunteers, Donors & Supporters

10 Kevin

Volunteer

Rating: 5

I'm a fix it Dad, no job to big or small. I fix broken toys, broken retail stores and offer help to "broken" people. When we found out about our son having Duchenne Muscualr Dystrophy, I came to realize I had something here I could not fix. Failure was not something I was used to. I simply had no answers, websites were filled with grim information, and our doctor was even worse. I shut down mentally. I lost my ability and motivation to fix anything else and then I lost my job. My sister-in-law found the PPMD web site and called my wife to tell her about it. My wife regestered us for the conference that was coming up and we went. I was still very wounded when we arrived, but the staff made us feel like we were in a safe harbor. They understood and were there to point us to the researchers and doctors who could answer our questions. We shared our stories and they listened to us. Some of them took pictures of our boys we were carrying and took them back to their labs, additional motivation for themselves. Pat Furlong the President of the PPMD lost both her sons to DMD, she lead me and so many others through the dark times and into a state where we not only understood what we were up against, but had a platform to build on. I learned to advocate, fundraise, and teach my story to other fathers who were in that dark place where I once lived. Pat, Kim and the entire staff of the PPMD are the kindest, most caring group of people you will ever meet. This group has worked hard and had many firsts by bringing all the experts together and building a model of what the standards of care are, as well as getting drug trials started. This is a full service organization. Kevin Smith

13

Volunteer

Rating: 5

Parent Project Muscular Dystrophy (PPMD) is an organization focused on improving the lives of those affected by Duchenne and Becker Muscular Dystrophy (DMD). They focus on DMD issues related to care, advocacy, research and education. PPMD has an exceptional Annual Conference that brings families and affected individuals together to learn from top researchers, clinicians and others in the community helpful to those living with DMD. Participants have much access to presenters and are able to ask questions after presentations and often one on one after. The PPMD website provides a wealth of information about many topics related to DMD. The organization hosts a community page linked to the main web page where friends and family members are able to meet for information and support. Another area PPMD has led the community is in Legislative Advocacy. PPMD and its family Advocates were successful in 2001 in passing the MD CARE Act which has dramatically improved NIH and CDC focus on the disorder. Advocates saw that the Act was reauthorized in 2008, a year when very little federal legislation passed, and Advocates have made great strides in increasing research for Muscular Dystrophy as a whole and education and outreach programs for DMD specifically. PPMD has reached out to other Volunteer Health Organizations who are interested in DMD both nationally and internationally. PPMD seeks to build these relationships to help the community and avoid duplication of efforts that waste resources. PPMD funds research that has the most promise to improving the lives of affected individuals. With advice from the organization's scientific adviser they have identified and funded potential opportunities that may slow the progression of DMD and/or improve the quality of life for those living with DMD. PPMD has developed a patient/family reported DMD registry. Aggregate information being collected may be useful for the research and clinical communities about DMD and the site will serve as a resource for identifying participants for future clinical trials. PPMD relies on fund raising and donations to support programs and works hard to provide to do this with as little administrative costs as possible.