Report of a TREAT-NMD/World Duchenne Organisation Meeting on Dystrophin Quantification Methodology

Aartsma-Rus A, Morgan J, Lonkar P, et al. Report of a TREAT-NMD/World Duchenne Organisation Meeting on dystrophin Quantification Methodology. Journal of neuromuscular diseases 2019;6:147-159   Abstract Representatives of academia, patient organisations, industry and the United States Food and Drug Administration attended a workshop on dystrophin quantification methodology. The aims of the workshop were to provide […]