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August 20, 2014
06:08 EDTFOLDAmicus Therapeutics reports positive Phase 3 data from Fabry study
Amicus Therapeutics announced positive 18-month data from its second Phase 3 study of the oral small molecule chaperone migalastat HCl - "migalastat" - in Fabry patients with amenable mutations. Study 012 compared oral migalastat to standard-of-care enzyme replacement therapies for Fabry disease. The co-primary outcome measures were the mean annualized changes in estimated glomerular filtration rate and measured GFR assessed by descriptive comparisons of migalastat and ERT over 18 months. Migalastat had a comparable effect to ERT on patients' kidney function as measured by the change in eGFR and mGFR. Levels of plasma lyso-Gb3, an important biomarker of disease, remained low and stable in patients with amenable mutations who switched from ERT to migalastat. Migalastat was generally safe and well-tolerated.
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February 24, 2015
08:33 EDTFOLDArgos Therapeutics hires Joan Winterbottom as Chief Human Resources Officer
Argos Therapeutics (ARGS) announced that Joan Winterbottom will join the company as chief human resources officer. Winterbottom most recently served as senior vice president of human resources at Amicus Therapeutics (FOLD).
February 23, 2015
07:22 EDTFOLDSunTrust to hold a conference
2015 Biotechnology and Pharmaceutical 1:1 Orphan Drug Day is being held in New York on February 23.

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