Rare Disease Day 2025
Today is Rare Disease Day!
Research opportunities for people affected by hepatic GSDs.
Find out about how to get involved in current hepatic GSD research studies.
Update on the Beam GSD1a programme.
Beam Therapeutics have put out a press release that includes an update on U.S. Food and Drug Administration clearance for their investigational new drug application for BEAM-301 in GSDIa.
Virtual AGSD-UK support.
Virtual Groups As children are returning to school, AGSD-UK will be holding a virtual group for parents or carers who might have questions around SEN, anxiety, or behaviour. If you would like to attend on the 9th September at 7.30pm please...
Update on Spark Therapeutics Pompe gene therapy programme.
Spark have provided the following community update on their Pompe gene therapy programme.