MENLO PARK, CA (February 28, 2013) The Myotonic Dystrophy Foundation (MDF) announced today that it has launched the Myotonic Dystrophy Family Registry (MDFR), a new online database designed to help both individuals living with myotonic dystrophy (DM), and the researchers and medical professionals studying the rare disease.
Press Release: MDFR Launch
“We intentionally designed the registry to be patient-driven and web-based,” said Molly White, executive director of MDF. “We want to collect a variety of information, beyond data on disease symptoms, to help support the development and implementation of clinical trials and to drive advocacy efforts focused on improving the circumstances of people living with DM. It’s important that we give people the opportunity to see what the rest of the community looks like and what they are experiencing. Our goal is to have at least 3,000 registrants, the ideal minimum number needed to help drive successful clinical trials.”
The MDFR focuses not only on statistically relevant demographic information, but also on additional patient-reported information, such as quality of life data. The MDFR is the first DM-focused registry that allows anyone registered to access the collected data on an anonymous basis. The MDFR will also provide access to information about clinical trials and studies.
The MDFR platform is provided by Patient Crossroads (PC), a leading international provider for rare disease registries. Patient Crossroads currently provides the registry platform for over 30 rare disease organizations around the world. The National Institutes of Health (NIH) recently selected Patient Crossroads through a competitive process as the lead registry vendor to establish a Global Rare Diseases Registry and Data Repository (GRDR) to collect patient-entered, anonymous information for clinical research.
The MDFR will complement the DM registry housed at the University of Rochester and other DM registries around the world, and is part of an NIH-funded registry standardization project.
“Because the MDFR is less focused on deep-data collection and does not capture complex data normally entered by clinicians, these other registries are critical to continuing disease discovery and basic research,” White added. “We encourage people living with DM to participate in all relevant registries, and have included links to other registries on the MDFR website.”
About Myotonic Dystrophy: Described as “the most variable of all diseases found in medicine”, myotonic dystrophy is an inherited disorder that can appear at any age and that manifests differently in each individual. The most common form of muscular dystrophy, DM affects somewhere between 1:3,000 and 1:8,000 people worldwide, and can cause muscle weakness, atrophy and myotonia, as well as problems in the heart, brain, GI tract, endocrine, skeletal and respiratory systems.
About the Myotonic Dystrophy Foundation: The Myotonic Dystrophy Foundation (MDF) is the world’s largest DM patient organization. Its mission is to enhance the lives of people living with myotonic dystrophy, and advance research efforts focused on finding treatment and a cure for this disorder through education, advocacy and outreach.
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