That is the theme of the data - consistent dystrophin production across all treated patients; consistent stabilization of 6MWT results for the ambulatory boys; and consistently pristine safety results. Anyone that is writing off these results as meaningless due to the small sample size just doesn't get it. When all the patients respond in a consistent manner, it doesn't matter whether the n is 12 or 120. AA is a slam dunk!
I don't think EFFICACY is the concern here to the FDA or anyone else. Its the potential adverse effects -- which may not have cropped up in a small patient population. BUT, FDA hasn't raised that point during their review of AA-signalling -- so, I consider the last FDA's minutes to be very bullish.