The first patient has been dosed in the FORTIS Phase 1/2 trial of AT845, a single-dose gene therapy for adults with late-onset Pompe disease (LOPD)
Avalglucosidase-alfa was awarded EAMS (Early Access to Medicines Status) in February 2021.
We are sad to report that AGSD-UK member Aldwyn Cooper has passed away.
The John Walton Muscular Dystrophy Research Centre in Newcastle is running a new study in Pompe disease.
The Pompe Support Team wanted to create a fun opportunity for children, as a show of support during lockdown.
Spark Therapeutics Announces First Participant Dosed in Phase 1/2 Study of Investigational Gene Therapy for Late-Onset Pompe Disease