CRANBURY, NJ, January 4, 2013 – Amicus Therapeutics today announced positive preliminary results from all 4 dose cohorts in a Phase 2 study (Study 010) to evaluate the safety and pharmacokinetic (PK) effects of the pharmacological chaperone AT2220 (duvoglustat HCl) co- administered with enzyme replacement therapy (ERT)A treatment that replaces the missing enzyme through IV infusion. for Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup that damages muscles and can affect breathing and, in some cases, the heart. (Myozyme® and Lumizyme®). Myozyme and Lumizyme (alglucosidase alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of the GAA enzyme., or recombinant human GAA enzymeA protein that helps the body carry out chemical reactions., rhGAA) are the first and only approved treatments for Pompe disease. Based on the Study 010 results, Amicus expects to initiate a repeat-dose clinical study in the third quarter of 2013.
To read full press release, please click here.