Sorry to slight you :-). Just remember a lot of the back and forth with Genisi and your right she did moderate (maybe she saw a trend coming in the data).
I got lucky (well I still did take a hit when I sold out a couple years ago). I thought the drug would work in Fabry just didn't see them getting enough near term financial benefit and I'm not a catalyst trader so stayed away.
I think we agree they had a tough task going up against ERT's. I guess the hope for them was finding a lot of adults who didn't want (or need to) go on ERT. I vaguely recall reading that the number of people with Fabry may be greatly underestimated. I don't know how well GSK could have done finding these people though.
I always thought Pompe was the key. I was (and perhaps even more so now) in the skeptical camp for their Pompe program after the tox in their monotherapy. I think they'll hit tox issue before they get the dose high enough to compete with newer Pompe programs in development.
Genisi actually became less pessimistic (or more optimistic) later on
Yes, less pessimistic. My words were "If so, I am less bearish on the monotherapy treatment than before*" (#msg-70875050), thinking FOLD might have found potential responders bearing certain 'amenable' mutations. But even so, and if the trend seen now (after 6 months) strengthen after 6 more months, I still don't see migalastat beats ERTs. I recall you were bearish on the combination with ERT (price, hard to show clinical benefit) while I thought they have a better shot to show clinical benefit with less frequent dosing. Still, I think we both are bearish on the commercial potential. Good for PLX's Fabry program that one player is out.
*From 3 years ago: "My thoughts were and still are, that chaperones will not deliver" (#msg-42150893)
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