Spark Therapeutics doses the first participant in Gene Therapy trial.
Spark Therapeutics Announces First Participant Dosed in Phase 1/2 Study of Investigational Gene Therapy for Late-Onset Pompe Disease
EMA accepts regulatory submission for avalglucosidase alfa.
Avalglucosidase alfa reaches its first important regulatory milestone
AGSD-UK attend NICE scoping workshop.
AGSD-UK working with NICE towards approval for new treatment for Pompe disease.
Pompe Support Team’s leaflet used in Australia!
Pompe Support Team’s Medical Overview leaflet is now being used in Australia.
Audentes Pompe Gene Therapy Trial News.
Audentes is now enrolling patients with late-onset Pompe disease in the Phase 1/2 clinical trial.
Support the call for new born screening.
New born screening has long been championed by AGSD-UK. Now you have an opportunity to support a larger national campaign.