Avalglucosidase-alfa was awarded EAMS (Early Access to Medicines Status) in February 2021.
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.
Has your child been diagnosed with Juvenile Myoclonic Epilepsy?
We are sad to report that AGSD-UK co-founder Ann Phillips has passed away shortly after contracting COVID-19 in her care home. She was 86.