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iwfal

06/26/17 8:53 AM

#212090 RE: mcbio #212089

What about their PPMO program? They have talked that up quite a bit. I believe it is set to enter clinic by end of this year. Do you think that can overcome the issues that you predict will plague etep and the other related more advanced exon skipping programs?



No idea. I don't routinely track SRPT for the obvious reason that, as the FDA documents revealed, they have a horrendous history of being very misleading. Also note that so far we have no results from the next gen oligos, which allow much lower dosing since they are better at staying at the target organs. (It'll be 3 or 4 years until we have big, long duration, RCT data for one of these drugs)

masterlongevity

06/26/17 1:59 PM

#212101 RE: mcbio #212089

also regarding $srpt, the truth is coming out regarding their deceptive drug pricing. as it turns out, their average price for an average weight pt is more like 750K-$1M per year. when you think of that price for a drug with questionable efficacy, it seems completely unethical. In addition to barking at insurance companies, parents should be knocking down $srpt door to get them to cut the price by 75% or more until they have a confirmatory trial completed.