A Concisely Recorded Ambulatory Assessment for Enhancing Real-World Outcomes Research in Duchenne Muscular Dystrophy: Development and Validation
A Concisely Recorded Ambulatory Assessment for Enhancing Real-World Outcomes Research in Duchenne Muscular Dystrophy: Development and Validation
At many centers and practices, the recording of standardized ambulatory assessments in DMD medical records is limited or inconsistent, which hinders retrospective learning from real-word data in this rare, progressive and life-limiting disease. In this study, we evaluated whether consistently recording a concise subset of ambulatory assessments, conducted as part of a complete assessment per care guidelines, could accurately quantify real-world disease progression for DMD research.
This poster was presented at AAN 2022, WMS 2022, and MDA 2023.
Authors: A. Mayhew, J. Signorovitch, V. Straub, C. Marini Bettolo, R. Muni-Lofra, A. Manzur, V. Ayyar Gupta, V. Selby, and F. Muntoni.