To me this paper sounds like its 2008 but with a single injection. Lot more efficacy over the current weekly dosing regime.  http://www.sciencedirect.com/science/article/pii/S0960896613008766 In 2008 we first saw the videos of GRDM models seeing the benefits of exon skipping, and now 5 years later we are almost on the verge of large scale trials for different exon skipping. The above paper talks…