Biotech Values

[ghmm]

SRPT:

I have no position in the stock. I read the article and thought it was well written. I've read other things that she has written/posted and think she is one of the more knowledgeable/credible posters. I think it would be worthwhile for longs to consider she may be right rather than resort to name calling. If one wants to be critical perhaps pointing out individual points would be received better without the name calling.

I invest in a fair amount of companies that are developing Orphan drugs. IMO I think they will need more data (I think a small Phase 3 trial of some sort). Admittedly I do not follow the company as closely as many of the passionate longs. Unfortunately (for those suffering from them) there are 6,000+ rare disease, many are fatal/debilitating, probably at least in the hundreds have/had trials and maybe a couple dozen have approved therapies. I can't think of one modern (so exclude drugs like Acthar in some indications) approved therapy with such little data. Unfortunately many patients did not survive or deteriorated while those therapies went through the regulatory/approval process. It does seem to hold a lot of promise but there are enough questions that I think FDA will want to see more data.