Posted on: 13 March 2026, source: Bezinga The 36-week data from the phase 3 study of DTX301 showed promising results, with a significant increase in urinary orotic acid levels. At week 36, DTX301-treated patients (n=18) demonstrated a 18% reduction in 24-hour plasma ammonia compared to placebo (n=19) and maintained average ammonia AUC0-24 in the normal range through week 36. Eight of nine patients with abnormal ammonia at baseline, despite optimal current drug treatment and diet restriction, rea
Ultragenyx's Gene Therapy Study Shows Ammonia Reduction In Rare Metabolic Disorder
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