Biotech Values

[ghmm]

SRPT:

Ok, so they approve it. And, assume they approve at roughly the same time 2 other drugs with overlapping patient pops, but much stronger datasets. It is very likely that most people will take Etep instead of the other drugs even though it has much less real data. And thus has a much better chance of failing future trials - I.e. They can't and don't really know what it works best at, if anything. There is a very real real world harm from approving drugs with essentially no data proving efficacy or helping decide where the efficacy is. It puzzles me that people forget this. False knowledge harms.

I'm not following where the harm comes in could you elaborate?

Not to bring up the old ERT argument again (and I am certainly not comparing the efficacy to ERT's) but one important finding that I think may be quite pertinent is that starting treatment very early in the disease and treating longer exaggerates the benefit.

A reasonable hypothesis. Suggest it needs proof. wink

For ERT's its proven. I am certain BMRN has long term data on Naglazyme treated patients and probably if I dig suspect I could find others (jq or genisi if she still monitors this board probably could find some quicker). More relevant to out discussion though this may bring up another area of disagreement in a different area never-the-less there are some (granted preclinical at least the ones I am aware of) papers on dystrophin production in mice and the conclusions support that. I think I have at least one on my hard drive if your interested (and don't already have it) I could try to find it this weekend sometime.