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.
Update on phase 3 study of DTX401.
Ultragenyx have provided the following community update on the phase 3 study of DTX401, their investigational gene therapy for the potential treatment of GSDIa. GSD1a Community...
International Pompe Day.
Find out about new Pompe information resources
Save the Date!
Main Event 2024