Outcome Measures and Natural History
The progression of many neuromuscular disorders (NMDs) remains poorly understood, and validated outcome measures are limited. To improve diagnosis, monitor disease progression, and evaluate treatment response, NMD4C is building a national framework to standardize outcome assessments, conduct natural history studies, and integrate patient-reported and imaging data. This work will support clinical trials, real-world evidence generation, and improved care for individuals living with NMDs.
Mission
To advance the understanding and management of NMDs in Canada through the development of validated outcome measures, natural history studies, and diagnostic tools that integrate clinical, imaging, and patient-reported data.
Objectives
Outcome Measure Training
To support standardized data collection and build national capacity, NMD4C and MDC organized two in-person training workshops focused on spinal muscular atrophy (SMA) outcome measures. These sessions brought together clinicians and evaluators from across Canada to align on best practices and strengthen consistency in outcome data collection.