Amicus Therapeutics and GlaxoSmithKline Announce Top Line 6-Month Primary Treatment Period Results From First Phase 3 Fabry Monotherapy Study
genisi was a bit pessimistic on the drug working and deserves credit for being so! I had sold out of my FOLD some years ago (at a loss) disappointed mainly with their Pompe program. The stock will likely get killed but perhaps some hope still for their co-administration.
In Study 011 patients with evaluable baseline biopsies, 13/32 (41%) in the migalastat HCl treatment group demonstrated a 50% or greater reduction in kidney interstitial capillary GL-3 after 6 months of study treatment versus 9/32 (28%) in the placebo group. This difference did not achieve statistical significance (p=0.3) according to the pre-specified primary endpoint analysis.
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