Update on Pompe Disease Treatment.
Avalglucosidase-alfa was awarded EAMS (Early Access to Medicines Status) in February 2021.
Aldwyn Cooper 1949 – 2021.
We are sad to report that AGSD-UK member Aldwyn Cooper has passed away.
Trial: Seroprevalence study in Pompe disease.
The John Walton Muscular Dystrophy Research Centre in Newcastle is running a new study in Pompe disease.
Winner announced of the PST Avatar Competition.
The Pompe Support Team wanted to create a fun opportunity for children, as a show of support during lockdown.
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