Jeff Harvey demonstrates the difficulties caused by the lack of step-free access on public transport in London.
The top eleven questions that patients, families and healthcare professionals want to be addressed in liver storage disease by researchers.
101 Top Tips for Pompe disease is now available in Portuguese.
The first patient has been dosed in the FORTIS Phase 1/2 trial of AT845, a single-dose gene therapy for adults with late-onset Pompe disease (LOPD)
Avalglucosidase-alfa was awarded EAMS (Early Access to Medicines Status) in February 2021.
We are sad to report that AGSD-UK member Aldwyn Cooper has passed away.