Recon: FDA approves Ultragenyx gene therapy for glycogen storage disorder

The FDA has approved Ultragenyx’s gene therapy for glycogen storage disease type 1a, providing a first-ever treatment option for patients aged 8 years and older with this rare metabolic disorder.

This Wire brief sits within Fusion42's coverage of Biotech, and 3 sources have reported it between 19 Aug 2026 and 20 Aug 2026.