Experts present key considerations in the design of rigorous preclinical studies in rodent models of muscular dystrophy, including animal model selection, assays and endpoints, and how your results can inform translation to the clinic.

In the muscular dystrophies (MD), the vigorous pace of basic and clinical discovery is generating a growing number of small molecule and genetic strategies towards the goal of a genetic cure or halting disease progression. In most cases, the path to the clinic requires a proof-of-concept study followed by an IND-enabling study in small animals to evaluate toxicity and estimate the starting dose.

This webinar will cover key considerations in the design and execution of rigorous pre-clinical efficacy studies in rodent models of MD. With a focus on Duchenne MD, we will consider the rodent models available, deciding on appropriate assays and endpoints, and how the results may inform the translation to the clinic.