This is quite puzzling. 9 patients in the placebo group achieved a 50% reduction in GL-3 after 6 months - on no treatment. Yet the absolute percent reduction in the placebo group was 6% vs. 41% on migalastat HCI? How does that math add up? Too bad the sample size is so small, as it appears 1 or 2 outliers skewed the result. Still holding hope for positive results next year, but I guess I can ignore FOLD for while. Paper loss for now.
The drug actually worked as you can see from the secondary endpoint analysis. ( median reduction of 41% in the migalastat HCl group versus a median reduction of 6% in the placebo group (p=0.093). The problem is the reading of the tissue biopies. The placebo effect should have been zero. I believe management referred to this possibility in the last presentation.