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)
Clinical Survey Study for GSD3.
Clinical Survey Study to Evaluate Biomarkers and Clinical Manifestations in Individuals With GSD3.
Update on Pompe Disease Treatment.
Avalglucosidase-alfa was awarded EAMS (Early Access to Medicines Status) in February 2021.
RCPCH &Us Rare Disease Survey.
RCPCH &Us have a survey for children & young people aged 13-25, who have a rare disease & live in the UK.
Aldwyn Cooper 1949 – 2021.
We are sad to report that AGSD-UK member Aldwyn Cooper has passed away.
Trial: Seroprevalence study in Pompe disease.
The John Walton Muscular Dystrophy Research Centre in Newcastle is running a new study in Pompe disease.