Parent Project Muscular Dystrophy Convenes Outcome Measures Meeting

20th Anniversary Conference Leads Off with Critical Discussions Aimed at Improving Clinical Trials in Duchenne Muscular Dystrophy

Jun 19, 2014, 14:30 ET from Parent Project Muscular Dystrophy

HACKENSACK, N.J., June 19, 2014 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD), the leading advocacy organization working to end Duchenne muscular dystrophy (Duchenne) will convene a pre-conference meeting of physical therapists, occupational therapists, and clinical coordinators who specialize in the research, assessment, longitudinal data collection, and treatment of people with Duchenne, June 25 and 26 in Chicago. This meeting will precede PPMD's 20th Annual Connect Conference, June 26-29.

PPMD Founding President and CEO, Pat Furlong, explains the origin of the meeting: "For the last several years, PPMD has brought together experts in Duchenne care to discuss gaps we have found in the clinical system. Recently, we held meetings with several biotech and pharmaceutical companies to explore barriers to clinical trials. Our discussions focused on three major areas of concern:  variability in care, the need for standardizing physical therapy evaluation of a child with Duchenne and recommendations for care, and the need for standardizing outcome measures for clinical trials. This year's pre-conference meeting will bring together top therapists from clinics around the country and start what we hope will be an ongoing dialogue about standardizing outcome measures. We are blessed to be a rare disease with a pipeline full of potential therapies. Now we need to make sure there are uniform parameters in place so that clinical trials in Duchenne are as effective and efficient as possible."

The goals of this meeting are to establish a platform for dissemination and discussion of the most up-to-date information about Duchenne in an effort to coordinate care and standardize the collection of outcome measures. The agenda will include discussions of magnetic resonance imaging and spectroscopy, strength, function, and respiration. These discussions will lay a foundation for outcome measures in all areas.

PPMD is hosting this meeting with the help of Claudia Senesac, PT, PhD, PCS from the University of Florida (Gainesville). PPMD has a long-standing history with the University of Florida – first supporting the investigation of MRI as a potential outcome measure and expansion of natural history studies for Duchenne with "Imaging DMD." PPMD initiated discussion with the University based on concerns expressed from meetings with industry partners. Dr. Senesac will help lead this effort to examine the nuances of measuring outcomes so that we can better standardize the way in which clinical trials are conducted.

This will be the first of several meetings that will provide training and standardization of outcome measures in Duchenne. PPMD will provide the Duchenne community, including therapists and clinicians unable to attend, with regular updates from these ongoing discussions.

To learn more about PPMD's extensive care initiatives, visit PPMD's website.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 3,500 live male births (about 20,000 new cases worldwide each year). Because the Duchenne gene is found on the X-chromosome, it primarily affects boys; however, it occurs across all races and cultures.

Duchenne results in progressive loss of strength and is caused by a mutation in the gene that encodes for dystrophin. Because dystrophin is absent, the muscle cells are easily damaged. The progressive muscle weakness leads to serious medical problems, particularly issues relating to the heart and lungs. Young men with Duchenne typically live into their late twenties.

Duchenne can be passed from parent to child, but approximately 35 percent of cases occur because of a random spontaneous mutation. In other words, it can affect anyone. Although there are medical treatments that may help slow its progression, there is currently no cure for Duchenne.

About Parent Project Muscular Dystrophy

Duchenne is a fatal genetic disorder that slowly robs young men of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our 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.

About ImagingDMD

ImagingDMD is funded by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and the National Institute of Neurological Disorders and Stroke (NINDS). The overall objective of this study is to validate the potential of noninvasive magnetic resonance imaging (MRI) and spectroscopy (MRS) to monitor disease progression and to serve as an outcome measure for clinical trials in Duchenne muscular dystrophy.

ImagingDMD is a joint effort between researchers at the University of Florida (UF), The Children's Hospital of Philadelphia (CHOP), Oregon Health and Science University (OHSU), Shriners Hospital for Children-Portland and the University of Pennsylvania (UPenn). Dr. Krista Vandenborne of UF, the study's director along with Dr. Lee Sweeney of UPenn, the study's co-Director, are leading a national group of experts in this effort. Persons interested in learning more about the study are encouraged to contact us.

Project Manager: Claudia Senesac (352) 273-6453.

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SOURCE Parent Project Muscular Dystrophy