Dystrophin 101 Webinar (March 2015)
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Dystrophin 101: Everything You Always Wanted to Know About the Duchenne Protein (and were not afraid to ask)
This webinar reviewed the basic biology of dystrophin (the gene, structure-function of the protein, its role in the dystrophin-glycoprotein complex, characteristics of different forms of dystrophin (both naturally occurring and drug-induced truncated forms), discussed how dystrophin is measured in both preclinical studies and clinical trials, and discussed the need for and role that dystrophin analysis play in clinical trials of novel agents designed to exert their therapeutic effects through an increase in dystrophin levels. We also discussed what is required in order to have dystrophin considered as a surrogate.
Speakers:
- Professor Francesco Muntoni, Chair of Paediatric Neurology and Director of the Dubowitz Neuromuscular Centre, University College of London
- John Porter, Ph.D., CEO, Parent Project Muscular Dystrophy