MDA Announces First U.S. Neuromuscular Disease Registry Report
MDA seeks to enhance and expand the registry in 2018
CHICAGO, Dec. 13, 2017 — The Muscular Dystrophy Association (MDA) today announced the release of the Highlights of the MDA U.S. Neuromuscular Disease Registry (2013-2016) report, which describes data collected during the pilot phase of the MDA U.S. Neuromuscular Disease Registry (“the registry”). The Highlights report is available for download via the U.S. Neuromuscular Disease Registry page on mda.org.
MDA launched its registry in 2013 to collect data on individuals with four diseases: amyotrophic lateral sclerosis (ALS), spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). These diseases were chosen based on the status of the therapy development pipeline at the time of the pilot launch and because each had in place formal “standards of care” — considerations that provided a unique opportunity to demonstrate how a registry could be used to measure the implementation of these care standards and their impact on patient outcomes. In the pilot phase, data was collected in 26 MDA Care Centers throughout the United States. Information collected in the registry is entered by physicians and/or study coordinators.
The goals of the U.S. Neuromuscular Disease Registry are to optimize the clinical management of individuals with neuromuscular diseases in order to extend survival and improve quality of life, and to help advance therapy development.
The Highlights of the MDA U.S. Neuromuscular Disease Registry (2013-2016) report shares demographic and disease-specific data for a subset of individuals seen in MDA-supported Care Centers that includes information about the average age participants were diagnosed, genetic diagnostic data, treatments used, common surgeries, nutritional interventions, ventilation needs, assistive devices, and how many individuals participated in clinical trials.
“The registry enables MDA to share information that can lead to a better understanding of the health of individuals who visit MDA-supported Care Centers across the United States,” said MDA President and CEO, Lynn O’Connor Vos. “On the heels of several recent breakthroughs in treatments for ALS, SMA and DMD, we are thrilled to be able to provide timely insights derived from registry data to serve as a valuable tool for researchers, clinicians and other key industry stakeholders who will be able to use it to innovate therapy development and transform clinical care for individuals living with neuromuscular disease.”
MDA’s disease registry is overseen with the highest level of protections for patients and their information and participation in the registry is voluntary. An independent committee called an Institutional Review Board (IRB) protects the rights and welfare of participants involved in the registry and ensures all research conducted is held to the highest ethical standards. Data from the registry used for research purposes is de-identified, meaning that the identity of the individuals enrolled in the registry is protected and not able to be connected to a particular individual’s clinical data.
“We are grateful for those individuals living with neuromuscular disease who participated in the pilot phase of the registry and for the commitment of the MDA Care Center sites and health care providers involved,” Vos said. “Without them our efforts to launch the registry, collect data and share key insights to standardize and optimize care for our families would not be possible. The registry is a means to improving quality of life, and MDA is committed to enhancing and expanding it in 2018.”
MDA is grateful for the many partners who make our mission possible including Biogen and Cytokinetics for their support of the registry.
MDA is leading the fight to free individuals — and the families who love them — from the harm of muscular dystrophy, ALS and related muscle-debilitating diseases that take away physical strength, independence and life. We use our collective strength to help kids and adults live longer and grow stronger by finding research breakthroughs across diseases; caring for individuals from day one; and empowering families with services and support in hometowns across America. Learn how you can fund cures, find care and champion the cause at mda.org.
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