I am very curious to see what their end-point will be. I would like it to be Keratan sulfate but I don't think the FDA would go for that as the primary end point :-). They said they have over 600 patients identified already! I would want to be very cautious since I lost a lot of faith in PEG-PAL being a blockbuster. If they succeed the treatment will have an incredible sales ramp given the 300-400k annual price (maybe even more I think some of the other ERT's are a bit higher).
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