Update on Pompe Disease Treatment.
Avalglucosidase-alfa was awarded EAMS (Early Access to Medicines Status) in February 2021.
Avalglucosidase-alfa was awarded EAMS (Early Access to Medicines Status) in February 2021.
RCPCH &Us have a survey for children & young people aged 13-25, who have a rare disease & live in the UK.
The John Walton Muscular Dystrophy Research Centre in Newcastle is running a new study in Pompe disease.
Eurordis key findings from a survey on patients’ and carers’
experience of medical care for their rare diseases.
Spark Therapeutics Announces First Participant Dosed in Phase 1/2 Study of Investigational Gene Therapy for Late-Onset Pompe Disease
BIMDG have provided a list of frequently asked questions about the vaccination programme for COVID-19