>> PTC's ph3 is to a large extent the very kids that SRPT and BMRN claim are patients in whom efficacy cannot realistically be measured. I agree that that provides some doubt over the BMRN and SRPT thesis.
That's simply not true if you look at PTCT ph3 inclusion criteria rather than their so called "conclusion" regarding >350m from ph2b trial that came from a sample including <7 yrs old, and more than 1/3 of >350m >7 yrs old actually were >450m. Their ph3 inclusion criteria has no upper limit on 6MWT, >150m with <80% predicted based on age and height, 7-16 yrs old - they will end up with many >350m, thus stratified by age > or < 9, 6WMT > or <350m. The <80% predicted limit would only get rid of maybe less than 10% of ph2b placebo. That's not much different from eteplirsen trial of 7-13 yrs old, 200-400m (+-10%) 6WMT. That's why Ohad correctly called himself as an observer compared to his writings on oncology. He would have been able to see these types of details in oncology trials.