AGSD-UK working with NICE towards approval for new treatment for Pompe disease.
A survey looking at the Covid-19 impact on wellbeing in families of children with rare neurodevelopmental and genetic disorders study
Audentes is now enrolling patients with late-onset Pompe disease in the Phase 1/2 clinical trial.
MetabERN has extended its survey deadline to June 21st 2020
Dr. Weinstein, long-time champion of improved diagnosis and treatment for hepatic GSDs due to move to Passage Bio as VP.
Survey on rare disease patients’ experience of COVID-19 will enable EURORDIS to measure the effect on lives and inform decision-makers.