Biotech Values

[poorgradstudent]

SRPT:

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.