
Trial: Seroprevalence study in Pompe disease.
The John Walton Muscular Dystrophy Research Centre in Newcastle is running a new study in Pompe disease.
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
Has your child been diagnosed with Juvenile Myoclonic Epilepsy?
AGSD-UK has teamed up with Vitaflo to learn more about how you live with liver GSD