Amicus Therapeutics announces MHRA approval for “Early Access” to their second generation ERT

Amicus Therapeutics announces MHRA approval for “Early Access” to their second generation ERT

Posted On: 08/06/2021 Recently Posted!

Today’s press release from Amicus Therapeutics, with quotes by Amicus CEO John Crowley, Dr Mark Roberts and Allan Muir, marks significant progress towards a second therapy for Pompe disease.

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:

Amicus Therapeutics Announces United Kingdom’s MHRA Grants Early Access to AT-GAA | Amicus Therapeutics (amicusrx.com)