Micro, your big cautionary IF seems to include hesitation about the efficacy of eteplirsen. I was just re-reading an MDA article about the 36-week results which said that the boys who were on 30 mg/kg for the first 24 weeks, then were on 50 mg/kg for the next 12 weeks did no better than the placebo group (placebo first 24, then 50mg for the next 12 weeks). http://quest.mda.org/news/extended-eteplirsen-treatment-benefits-walking-dmd
What that suggests to me is that at 30mg/kg, there is not much strength effect, even though dystrophin production is relatively high at 24 weeks. It will be interesting to see what the biopsy shows in terms of dystrophin production at 50mg. If it's way higher than at 30mg, then I will breathe a big sigh of relief; otherwise, not so sure.