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New Home-Based Assessment for Duchenne Muscular Dystrophy

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Emmes today announced that Casimir’s research on the development of the Duchenne Video Assessment (DVA) scorecards was published in PLOS ONE. Emmes acquired Casimir in March 2022, and the group is now part of Emmes’ Rare Disease Center, Orphan Reachâ„¢.

The DVA is a novel, home-based clinical outcome assessment that measures ease of movement among those with Duchenne muscular dystrophy (DMD) through identification of compensatory movement patterns.  Caregivers video record patients performing specific movement tasks at home using a secure mobile application.  The paper describes the development of the scorecards that DVA-trained physical therapists use to score the videos.

Casimir’s Marielle Contesse, research scientist, was the lead author, with Mindy Leffler, co-founder and president, and Christine McSherry, co-founder and chief executive officer, among the other co-authors.

A previous paper in Muscle & Nerve, authored by Contesse, Leffler and other Casimir researchers, presented research findings that demonstrated that the DVA is a reliable and valid tool for measuring ease of movement as an indication of disease severity in patients with DMD. The latest paper in PLOS ONE details the development of the DVA scorecards.

The PLOS ONE paper outlines the scorecard development research conducted with physical therapists who have extensive experience evaluating patients with DMD. The researchers used a two-round modified Delphi process to gather expert opinion on the scorecards. During the Delphi process, the physical therapists evaluated the scorecards for all 15 movement tasks. The movement tasks are activities that patients typically do at home, such as walking, climbing stairs, putting on a t-shirt, and eating. After two rounds of review and revisions, the expert panel confirmed that the compensatory movement criteria included in the DVA scorecards were understandable, comprehensive, and clinically meaningful.

“The DVA provides a window into the way patients with Duchenne move and function in their home environment,” said Marielle Contesse, “and our scorecards provide a standardized way for physical therapists to identify compensatory movement patterns. The at-home setting allows us to assess an even wider range of day-to-day movements. It also eliminates the need for patients and families to travel long distances to clinical trial sites.”

She added, “Ultimately, we hope that the DVA will be able to detect changes in patient function in a shorter period of time than existing outcome measures. This could potentially reduce the time that patients spend participating in a clinical trial.”

Emmes Chief Executive Officer Dr. Christine Dingivan noted, “This research provides the foundation for using Casimir’s Duchenne Video Assessment to widen opportunities for identifying disease progression and treatment options. We’re also encouraged by the potential use of the DVA to assess other muscular and mitochondrial diseases.”

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