Pretty amazing that it has come to this. Here is a drug that ostensibly produces less than 0.9% of normal dystrophin, in a manner that is statistically significant only if you set some of the baseline values at 0 rather than the lower limit of detection.
The fact that we're even discussing these data as part of the basis for approval is a testament to the lobbying efforts of the families.
With this upcoming analysis, I think they're supposed to be analyzing before and after samples from the same participant. If they do a paired analysis, minuscule "increases" in dystrophin are likely to be stat sig.
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