Success of preclinical drug trials using reliable and reproducible endpoints in mouse models of neuromuscular diseases


Preclinical efficacy evaluation in mouse models of human diseases is an important component of drug development. It has been reported that phase II clinical trial success rates have fallen significantly in recent years, with a lack of efficacy being the most frequent reason for failure. These failures could be due to 1) the poor predictive power of disease models, 2) questionable targets, 3) lack of rigor in preclinical trial design, 4) poor control for potential bias, or 5) variable reporting standards.


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