FDA grants AT-GAA breakthrough therapy in research to be identified as used to treat late-haired Pompeii disease

Pompeii disease is a genetic lysosome setic saclic disorder caused by the absence of acid-alpha-glucosinase (GAA)AT-GAA is the first to be identified as Pompeii diseasethe(http:// of theFDArecently, Amicus Therapeuticscompany(http://announced that the U.SFDA has awarded it a therapeutic AT-GAA breakthrough therapy for the treatment of late-night hair-dosicdiseaseon AT-GAAAT-GAA is a combination therapy developed by AmicusIt has two active ingredients, one is ATB200, a unique recombinant human GAA (rhGAA) proteaseIts carbohydrate structure is optimized to enhance the ability to be absorbed by cellsATB200 can replace the function of THE missing GAA in Pompeii patientsanother active ingredient is AT2221, a pharmacological companionGAAs produced by Pompeii patients themselves are usually degraded by a structural mutation The function of AT2221 is to combine with the patient's own GAA to improve their stability and thus maintain gaA levels 　　Amicus is a bio
pharmaceutical (http:// company dedicated to developing innovative treatments for rare diseases