Ultragenyx Announces U.S. FDA Acceptance and Priority Review of the Biologics License Application (BLA) for DTX401 AAV Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa)

Detailed price information for Ultragenyx Pharmaceu (RARE-Q) from The Globe and Mail including charting and trades.
Nature

Glycogen Storage Diseases And Polyglucosan Body Disorders

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 ...
Nature

Glycogen Storage Diseases

Glycogen storage diseases (GSDs) comprise a heterogeneous group of inherited metabolic disorders that impede the normal synthesis or degradation of glycogen, resulting in diverse clinical phenotypes.
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)

At Week 96 participants experienced even greater reductions in daily cornstarch intake while maintaining low levels of hypoglycemia, improved levels of euglycemia and improved fasting tolerance Phase ...
sciencex

World-first gene editing trial for rare genetic disease

In a global first, UConn Health, in collaboration with Connecticut Children's, has dosed a patient in a groundbreaking clinical trial testing gene editing technology to treat glycogen storage disease ...