Extension of Early Access to Medicines Scheme for trial Pompe drug.
An update on avalglucosidase alfa.
CureGSD1b Research Ready Data Collection Plan.
The CureGSD1b Research Alliance is working extensively on a full understanding of the research landscape related to GSD1b at the moment.
Progress on NICE assessment of new treatments.
An update on the appraisal of treatments for Pompe by NICE.
Lived experience advisory panel.
Could you represent the rare experience on this new venture from The Patient Association?
Whole Genome Sequencing for newborn screening.
Genomics England have released findings that show the public are generally supportive of using whole genome sequencing for newborn screening.
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.