The Pompe community has been following progress of this drug development since their early studies into pharmacological chaperones, first published in 2007.
Early access to this treatment through EAMS should help a small number of Late-Onset Pompe adults (18+) in the UK who are declining on the current therapy. The process of receiving AT-GAA will involve a short period of fasting, followed by taking a chaperone in pill form, before initiating the ERT infusion in the usual way.
Click below to read the press release: