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Amicus Therapeutics Inc FOLD

Amicus Therapeutics, Inc. is a biotechnology company, which is focused on discovering, developing, and delivering novel medicines for rare diseases. Its two marketed therapies are Galafold, the first oral monotherapy for people living with Fabry disease who have amenable genetic variants, and Pombiliti + Opfolda, a novel treatment designed to improve uptake of active enzyme into key disease relevant tissues for adults living with late-onset Pompe disease. As an orally administered monotherapy, Galafold is designed to bind to and stabilize an endogenous alpha-galactosidase A (alpha-Gal A) enzyme in those patients with genetic variants identified as amenable in a Good Laboratory Practice (GLP) cell-based amenability assay. Pombiliti + Opfolda consists of a uniquely engineered rhGAA enzyme, cipaglucosidase alfa-atga, with an optimized carbohydrate structure to enhance lysosomal uptake, administered in combination with miglustat that functions as an enzyme stabilizer.


NDAQ:FOLD - Post by User

Bullboard Posts
Post by osirus1on Oct 05, 2015 8:44am
225 Views
Post# 24162527

$FOLD upgraded $CTRV $AVXL $TBPH & $ASTI

$FOLD upgraded $CTRV $AVXL $TBPH & $ASTI$FOLD upgraded $CTRV $AVXL $TBPH $ONCE & $ASTI I like my US biotechs today all have good news out. Putting them on watch as the rising seas raises all ships. $SIEN a short today
Bullboard Posts