Biotech Values

[jq1234]

I am not against the drug. I just think the efficacy is limited, thus limited market potential. I agree with your assessment about those newly diagnosed with no to mild symptoms could be on the drug if they decide to be on therapy for a certain period of time. I just don't see it being like ERT where patients would be on for the rest of their lives with increased dosing with weight gain over time - that's where commercial success comes from for such small patient population.