Abstract
Promising therapeutic interventions for muscular dystrophy are rapidly increasing, leading to an increased demand for pre-clinical testing in mouse models of muscular dystrophy. Pre-clinical efficacy and toxicity assessments are critical steps in moving potential therapies from the bench to patient bedside. Currently, different mouse models, treatment protocols, age groups and end points are used to assess the drug efficacy in mice. Despite extensive research and pre-clinical testing there is no consensus regarding the most appropriate pre-clinical trial design and end-points (clinical, functional, histological, and biochemical) for muscular dystrophy mouse models.
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