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.
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...
Cipaglucosidase alfa (Pombiliti®) is accepted for use within NHS Scotland.
Cipaglucosidase alfa (Pombiliti®) is accepted for use within NHS Scotland The Scottish Medicines Consortium has completed its assessment of cipaglucosidase alfa and has advised that this offers an additional treatment choice as a long-term enzyme replacement...