Ultragenyx Announces U.S. FDA Approval of GENGLYCOS™ Gene Therapy, the First-Ever FDA-Approved Treatment Designed to Treat the Underlying Cause of Glycogen Storage Disease Type Ia (GSDIa)
FDA approval of first-ever gene therapy targeting GSDIa’s underlying cause marks a major regulatory milestone.
What changed
FDA approval of first-ever gene therapy targeting GSDIa’s underlying cause marks a major regulatory milestone.
Why it matters
FDA approval of first-ever gene therapy targeting GSDIa’s underlying cause marks a major regulatory milestone. — impact context not separately stored; see What changed.
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Aug 20, 2026 05:44 AM ET