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Breakthrough Closer: Ultragenyx’s DTX401 Gene Therapy Receives FDA Priority Review for Rare Sugar Disorder

Ultragenyx’s one-time gene therapy DTX401 just got FDA Priority Review for Glycogen Storage Disease Type Ia (GSDIa). Possible approval by August 23, 2026 – the first treatment that could fix the root cause instead of just managing symptoms.

Breakthrough Closer: Ultragenyx’s DTX401 Gene Therapy Receives FDA Priority Review for Rare Sugar Disorder
"The Ultragenyx biomanufacturing plant in Bedford, Massachusetts". Credit: Jonathan Wiggs/The Boston Globe/Getty Images
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