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jq1234

07/23/12 10:56 PM

#145932 RE: mcbio #145931

To me, it is important to have some idea how MUCH better the combination with ERT is than ERT alone in some types of clinical endpoints. It is easy to say monotherapy succeeded or failed, but difficult to say from single arm trial how much better the combination with ERT is than ERT alone. Ok, increased certain enzyme activity 1.5-fold or 2-fold, how does that translate into clinical benefit? So far we only saw partial data from drug-drug interaction study. Without some clinical endpoint data, it is very difficult to value FOLD IMO.