McArdle walking courses open
Full details of the 2025 McArdle walking courses are now available, and we are open for bookings.
Full details of the 2025 McArdle walking courses are now available, and we are open for bookings.
Today is Rare Disease Day!
Find out about how to get involved in current hepatic GSD research studies.
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.
Spark have provided the following community update on their Pompe gene therapy programme.