urea cycle disorders

halfway there
Biotech

Ultragenyx gene therapy lessens ammonia levels in ph. 3

One-time treatment with DTX301 led to 18% lower ammonia levels compared to placebo at 36 weeks, hitting one of the trial's two key endpoints.
Darren Incorvaia Mar 12, 2026 11:33am
Gene editing therapy patient KJ with Drs Ahrens-Nicklas and Musunuru

Boy with rare disease 'thriving' after 1st custom gene therapy

May 15, 2025 1:00pm