HACKENSACK, N.J., Aug. 12, 2015 /PRNewswire-USNewswire/ -- DuchenneConnect, led by Parent Project Muscular Dystrophy (PPMD) has been approved for a three-year, $1.4 million funding award by the Patient-Centered Outcomes Research Institute (PCORI) as part of the second phase of the development of PCORnet, the National Patient-Centered Clinical Research Network.
This funding award continues PCORI's support for DuchenneConnect's participation in PCORnet, a large, collaborative research initiative designed to link researchers, patient communities, clinicians, and health systems in productive research partnerships that leverage the power of large volumes of health data maintained by the partner networks. PCORnet will enable the nation to conduct clinical research more quickly and less expensively than is now possible and will ensure that research focuses on the questions and outcomes that matter most to patients and those who care for them.
PCORI provided close to $1 million in funding to DuchenneConnect during the first phase of PCORnet's development, which began in the spring of 2014. A model for other rare disease registries, DuchenneConnect successfully achieved Phase I goals of patient and caregiver engagement and governance, enhanced data collection, and rigorous but streamlined informed consent. DuchenneConnect successfully engaged the Duchenne and Becker communities on several projects throughout Phase I, including the identification of research priorities.
As one of 34 health data networks that together make up this national clinical research resource in Phase II, DuchenneConnect is contributing expertise and resources focused on Duchenne and Becker muscular dystrophies – rare, progressive and debilitating disorders. DuchenneConnect will continue to collaborate with PatientCrossroads and Geisinger Health Systems to achieve Phase II milestones, and with the University of California, Los Angeles through implementing shared priorities. The new funding award will support DuchenneConnect's continuing efforts to develop robust, mutually beneficial relationships with other networks within PCORnet where the needs and priorities of patients and caregivers are actualized through collaborative research and decision support tools.
Holly Peay, PhD, a Co-PI of the DuchenneConnect Registry Network stated, "DuchenneConnect will continue to contribute to PCORnet as a network that is directly accountable to patients and caregivers; as a network with years of collaborative research experience with academia, industry, patients, and families; in our emerging capacity to integrate electronic health record data; and most importantly as an engaged network responsive to the needs of our stakeholders."
"We're pleased that DuchenneConnect has been approved for this funding support to continue our productive mutual efforts to build what we intend to be a premier national resource for conducting high-quality, patient-centered clinical research," said PCORI Executive Director Joe Selby, MD, MPH.
The funding award to DuchenneConnect has been approved by PCORI's Board pending completion of a business and programmatic review by PCORI staff and negotiation of a formal award contract. PCORI is an independent, non-profit organization authorized by Congress in 2010 to fund research that will provide patients, their caregivers and clinicians with the evidence-based information they need to make better-informed healthcare decisions. PCORI is committed to continuously seeking input from a broad range of stakeholders to guide its work. More information is available at www.pcori.org.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 3,500-5,000 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 people 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 people 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 people with Duchenne live longer, stronger lives. We will not rest until every person with Duchenne has a treatment. 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