Eurordis H-Care Survey of rare diseases results.
Eurordis key findings from a survey on patients’ and carers’
experience of medical care for their rare diseases.
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
COVID-19 Vaccine FAQ’s.
BIMDG have provided a list of frequently asked questions about the vaccination programme for COVID-19
Seeking Lafora patients: Angelina’s story.
Has your child been diagnosed with Juvenile Myoclonic Epilepsy?
How are you living with a liver GSD?
AGSD-UK has teamed up with Vitaflo to learn more about how you live with liver GSD
EMA accepts regulatory submission for avalglucosidase alfa.
Avalglucosidase alfa reaches its first important regulatory milestone