Outcome measures and clinical assessment instruments in neuromuscular disease

Abstract

This chapter reviews issues which result from consideration of the optimal outcome measures. By its nature, the process of measurement requires reducing a dynamic process (the patient dealing with their disability) to a 2-dimensional snapshot (the patient's score at one point in time) that will be used to reflect a patient's condition. Measurement is usually an imperfect process, especially when relatively small treatment effects are expected. Multiple examples of available treatment instruments will be reviewed with an emphasis on their benefits and limitations for future use.