Audentes Pompe Gene Therapy Trial News.
Audentes is now enrolling patients with late-onset Pompe disease in the Phase 1/2 clinical trial.
Audentes is now enrolling patients with late-onset Pompe disease in the Phase 1/2 clinical trial.
AGSD-UK has appointed Julie Jackson to a temporary post to address a large increase in requests for help with state benefits.
New born screening has long been championed by AGSD-UK. Now you have an opportunity to support a larger national campaign.
Mendelian, working to reduce diagnostic delay, and thus improve quality of life, in rare diseases including glycogen storage disease.
During 2019 the rate of new registrations with AGSD-UK increased by 26% over the previous year. We consider the reasons why.