Online Resource Links Patients With Researchers, Clinicians
MIDDLETOWN, Ohio, Jan. 23 /PRNewswire-USNewswire/ -- 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 the one year anniversary of DuchenneConnect, a central online arena linking the resources and needs of the Duchenne and Becker muscular dystrophy community (those living with muscular dystrophy, their families, and caregivers) and the provider community (clinical care providers, policymakers, industry professionals, and the medical research fields).
DuchenneConnect provides resources to help individuals with Duchenne and Becker muscular dystrophy and their families understand the steps that may be required to confirm the diagnosis and the benefits and limitations of genetic testing. Information is available for those who may need to be retested and for those with a new diagnosis. The program works with regional and local resources where possible and connects participants to information about new treatments, trials, and services such as genetic counseling and genetic testing.
With over 1,300 registered patients and families, and 188 providers, Ms. Furlong has been thrilled with the quick growth of this still relatively new resource in muscular dystrophy. "Families affected by Duchenne and Becker muscular dystrophy do not always receive a proper diagnosis; fewer receive the most up-to-date treatments for these diseases. DuchenneConnect has given families across the country access to top researchers and clinicians in the field, as well as notification about clinical trials that may help their child. Gathering the community into a registry with global partnerships provides crucial information to researchers and clinical investigators, and may further accelerate therapeutic discoveries. To see what was just the seed of an idea of a group of passionate leaders in the Duchenne community, blossom into a thriving online resource, is truly phenomenal. We may be many years from knowing the direct affect of the early intervention DuchenneConnect provides, but I believe it will prove an extremely positive and crucial tool."
Early in 2007, a group of thought leaders in the Duchenne muscular dystrophy community, led by PPMD, began discussing the need for a new kind of resource that would connect and serve the needs of the entire community. What they envisioned was a central hub that would bring together those living with the disease, along with their families, friends and caregivers, to connect them with medical research, clinical care, clinical trials and each other. At the same time, it would also be a resource for industries with an interest in Duchenne, allowing access to a database of information provided by patients and their families --information that could prove vital to advances in care and treatment. This resource, a breakthrough multidirectional information portal for Duchenne and Becker muscular dystrophy, would help connect these stakeholders, while providing them with resources never before available in one place. Today, DuchenneConnect is a robust and cutting-edge website and registry meeting the goals and expectations of its creators.
DuchenneConnect is a collaboration of Centers for Disease Control and Prevention (CDC); Innolyst, Inc.; Emory University, Department of Human Genetics; National Institutes of Health (NIH) Office of Rare Diseases: Collaboration, Education and Test Translation (CETT) Program; National Center for Biotechnology Information (NCBI); and PPMD and supported by advisory experts throughout the neuromuscular community. It is managed and operated by DuchenneConnect Registry Coordinator, Vanessa Rangel Miller, MS, CGC, at Emory University.
Ms. Rangel Miller, who along with Ms. Furlong has been with DuchenneConnect since its inception, stresses the incredible influence the site is having on the medical community, and more specifically rare diseases: "DuchenneConnect is leading the way by providing a forum to unite patients with medical and research communities. The contribution of patient information to the registry has been an asset to a variety of clinical organizations, a trend which we predict will continue as the registry grows. Given its novel approach, DuchenneConnect has also become a model resource and registry emulated by other rare disease communities."
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.
To explore DuchenneConnect and experience the breadth and depth of its resources, as well as to see a complete list of contributors and advisors, please visit www.duchenneconnect.org.
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.
Launched in 2008, DuchenneConnect links the entire Duchenne and Becker community -- those living with Duchenne or Becker and their families, medical/research professionals, and the medical/pharmaceutical industry, and brings participants the latest information about current treatments and clinical trial opportunities. For more information, visit www.duchenneconnect.org or call 404.778.0553.
SOURCE Parent Project Muscular Dystrophy