Researchers at Freiburg University Hospital along with international partners are conducting a study on women’s health and pregnancy in hepatic GSDs.
If you are interested in taking part in this important study you can find out more here.
The U.S. Food and Drug Administration (FDA) has approved DTX401, a gene therapy developed by Ultragenyx for the treatment of GSDIa. Find out more here:
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.