Biotech Values

[ghmm]

Thanks again for the details here and on twitter, I learned a few things today!

I didn't change my position in SRPT though I acknowledge the risks. I just like to point out that besides 6 minute walk the continued ambulation (of all but the two who lost it early) and lung function measures also help to support the data package. As BMRN CEO likes to say I think the totality of the data in the light of no approved agents and a (extremely) strong parent lobbying effort will make the hurdle about as low as possible. The safety data from on-going studies may be enough to help reduce the risk of approving a drug with what may be modest efficacy.