Considerations for Rigorous Pre-clinical Studies in Murine Muscular Dystrophy

Considerations for Rigorous Pre-clinical Studies in Murine Muscular Dystrophy


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 covers 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 consider the rodent models available, deciding on appropriate assays and endpoints, and how the results may inform the translation to the clinic.

Key Learning Objectives Include:

  • Considerations when designing a pre-clinical efficacy study in rodent models of muscular dystrophy
  • Choosing animal models, assays and endpoints
  • How your results can inform the translation to the clinic

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Sharon Hesterlee, PhD
EVP, Chief Research Officer
Muscular Dystrophy Association

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.

She has been involved in numerous efforts to remove barriers to therapy development for rare disease and foster interactions between patient advocacy groups and industry. Dr. Hesterlee has served on the governing board of the Health Research Alliance, the NINDS Council, and the Department of Health and Human Services’ Muscular Dystrophy Coordinating Committee. She currently serves on the Congressionally Directed Medical Research Program’s Duchenne Muscular Dystrophy Programmatic Review Panel. She received her Ph.D. in neuroscience from the University of Arizona in 1999.

Ramzi Khairallah, PhD
Co-Founder & President

Dr. Ramzi Khairallah’s study and professional work has been closely focused on muscle function and dysfunction. He obtained his Ph.D. in molecular medicine from the University of Maryland under the late Dr. William Stanley. He has helped develop key procedures in the field and has published widely on muscle function, particularly on the cellular level, in journals such as Science Signaling, Nature Communication, and the Proceedings of the National Academy of Sciences a. He is co-inventor with Dr. Ward on several patents for therapeutic intervention in muscular dystrophies. Dr. Khairallah co-founded Myologica in 2014 with Dr. Ward and has since helped numerous companies with their pre-clinical neuromuscular interventions programs.

Chris Ward, PhD
Associate Professor, University of Maryland School of Medicine;
Chief Scientific Officer, Myologica

Dr. Christopher Ward has spent almost 30 years studying physiology and muscle function from the whole animal to the cellular level and has developed or helped develop many of the procedures now used in the study of muscle fiber. He holds a Ph.D. in physiology from the Virginia-Maryland Regional College of Veterinary Medicine. Along with W. J. Lederer and B. P. Prosser, he is co-inventor and holder of the patent for MyoTakᵀᴹ, the muscle cell adhesive. He and Dr. Khairallah have several patents for therapeutic intervention in muscular dystrophies. Dr. Ward has published numerous articles focused on muscle function and dysfunction in journals such as Science, Nature Medicine, and PNAS. He is currently a tenured professor at the University of Maryland.