Ultragenyx's Gene Therapy Study Shows Ammonia Reduction In Rare Metabolic Disorder

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