Replies to post #67496 on Biotech Values

[ghmm]

Thanks for the link. I should be more up on Pompe but my candidates (BMRN and FOLD) are a few years away.

[ghmm]

I read through the briefing doc (thanks again). It seems to give more then enough wiggle room for an interim solution. Something like Genzyme continues to provide drug (and charge for it) while needing to conduct another trial comparing it to the 160L with more younger patients.

I believe FVC was used in TKT/Shire's Hunter trial. I know it mentioned 6MW being the standard for Pompe. Do you have strong thoughts on one end-point vs. another in Pompe or do you feel surrogates aren't good predictors of longer term outcome?