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.
専門家の見解：
Christopher Ward
PhD
Ramzi Khairallah
PhD
Sharon Hesterlee
PhD
Dr. Hesterlee has over 20 years of experience in neuromuscular research in both the nonprofit and industry space. She has served as head of research for Parent Project Muscular Dystrophy, the Myotonic Dystrophy Foundation, the Association for Frontotemporal Degeneration and the Muscular Dystrophy Association’s MDA Venture Philanthropy. Dr. Hesterlee has also served as project lead for rare disease gene therapy programs at Pfizer, Inc., as Chief Executive Officer of Lion Therapeutics, and as Executive Vice President and Head of the Neuromuscular Division of Askbio Inc.
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