Jay S. Charleston

TL;DR: The present study used a double‐blind placebo‐controlled protocol to test eteplirsen's ability to induce dystrophin production and improve distance walked on the 6‐minute walk test.

TL;DR: This study provides Class I evidence that golodirsen is safe and Class IV evidence that it induces exon skipping and novel dystrophin as confirmed by 3 different assays.

TL;DR: This study provides Class II evidence of the muscle cell penetration, exon skipping, and induction of novel dystrophin expression by eteplirsen, as confirmed by 4 assays.

TL;DR: Despite the loss of ambulation, other markers of disease progression remained relatively stable in the eteplirsen-treated twin patients and were similar to those of the ambulatory patients.

TL;DR: The pathology of D MD and BMD is highlighted, animal models of DMD and B MD are discussed, and dystrophin biomarker quantitation is described, with several image analysis approaches, including a new automated method that evaluates protein expression of individual muscle fibers.