Glycogen storage disease type III (GSD III) is a rare genetic disorder resulting from mutations in the AGL gene, which encodes the glycogen debranching enzyme (GDE). This enzyme is vital for the ... Glycogen storage diseases (GSDs) constitute a group of inherited metabolic disorders that impair normal glycogen synthesis or degradation, resulting in the accumulation of structurally abnormal ...
BioWorld: Novel AAV-based gene therapy for liver glycogen storage disease type IX presented Liver glycogen storage disease type IX (GSD IX) accounts for 25% for all GSD cases, with a prevalence of 1 out of 100,000 patients. GSD IX is caused by deficiency in phosphorylase kinase (PhK), which ... Novel AAV-based gene therapy for liver glycogen storage disease type IX presented Seeking Alpha: Ultragenyx Announces Positive Longer-term Data from Phase 3 Study of DTX401 AAV Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia (GSDIa) Ultragenyx Announces Positive Longer-term Data from Phase 3 Study of DTX401 AAV Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia (GSDIa) Business Insider: Beam Therapeutics Presents Preclinical Data Highlighting Utility and Durability of BEAM-301 to Correct a Glycogen Storage Disease Type I Deficiency Disease-Causing Mutation at ... Beam Therapeutics Presents Preclinical Data Highlighting Utility and Durability of BEAM-301 to Correct a Glycogen Storage Disease Type I Deficiency Disease-Causing Mutation at ...
