Wednesday, October 10, 2018 4:23:43 PM
Severe allergic reactions to ERT for FD are uncommon but there are a small number of patients with severe allergic reactions who either have to stop ERT or have to take premedications such as hydrocortisone, which have their own adverse effects. While an effective oral alternative would be very useful in patients who cannot tolerate ERT, it is unlikely that migalastat can fill that role in that the more severe infusion reactions often
occur in patients with the more severe mutations (e.g., null mutations) and these mutations are not usually amenable to chaperone therapy. Thus, the issue of an effective treatment alternative for patients who cannot tolerate ERT is likely to remain an unmet need even with the availability of migalastat.
Protalix really claims for 50%+ of Fabry market has M/C $84M and Amicus $2.25B.It's absolutely crazy. We have already lost most of the recent SP rise.Apparently today the main players are shorts and penny flippers.For me, this is still a waiting game for at least one more year.
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