Progress on NICE assessment of new treatments.
An update on the appraisal of treatments for Pompe by NICE.
Step-Free Access in London – BBC Politics Show
Jeff Harvey demonstrates the difficulties caused by the lack of step-free access on public transport in London.
Liver Glycogen Storage Disease Top 11 Questions.
The top eleven questions that patients, families and healthcare professionals want to be addressed in liver storage disease by researchers.
Pompe book is going global!
101 Top Tips for Pompe disease is now available in Portuguese.
First Pompe patient dosed in FORTIS Trial of AT845.
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)
Update on Pompe Disease Treatment.
Avalglucosidase-alfa was awarded EAMS (Early Access to Medicines Status) in February 2021.