Ultragenyx Announces FDA Clearance of Investigational New Drug (IND) Application for UX053.
Ultragenyx Announces FDA Clearance of Investigational New Drug (IND) Application for UX053, an mRNA for the Treatment of GSD3
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.
Trial: Seroprevalence study in Pompe disease.
The John Walton Muscular Dystrophy Research Centre in Newcastle is running a new study in Pompe disease.