MITT has been widely used in FDA submissions!
Single site is a concern, but the fact it is at a highly visible site, plus the dystrophin production at the 2 UK sites in the phase I alleviates much of these concerns.
Some ...... will say anything to fortify their negative positions.
He is obviously one of those people!
These newbies are misleading..... Just when you think they they may have a valid argument, turns out they just have not done their homework or reviewed they history of the science/results and data. Like dealing with a non linear thinker, AKA Attention Deficit Disorder investors expect you to fill in the blanks..
I think you also have to look at the very small patient population as being a contributing factor, and thus making it somewhat of non-issue, for the single site and the fact that the disease is deadly with no other real treatment options. If this was a drug treatment something that was not deadly, had a big patient population and there were other treatments then the argument would be a good one. That is not the case here.