Andrew Wakelin has an article about his McArdle’s walking courses in the latest National Trust magazine
Making the Unseen Seen: Rare disease and the lessons learned from the COVID-19 pandemic.
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, an mRNA for the Treatment of GSD3
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)