AGSD-UK’s Andrew Wakelin featured in National Trust magazine.
Andrew Wakelin has an article about his McArdle’s walking courses in the latest National Trust magazine
Rare disease and the lessons learned from the COVID-19 pandemic.
Making the Unseen Seen: Rare disease and the lessons learned from the COVID-19 pandemic.
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.
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)