Ultragenyx Pharmaceutical RARE reported positive updates from the Stage 1 cohorts of its phase I/II/III Cyprus2+ study ...

A minority of patients taking Ultragenyx Pharmaceutical’s Wilson disease gene therapy UX701 have come off standard-of-care ...

In patients who have tapered off standard-of-care, non-ceruloplasmin bound copper (NCC) has stabilized to normal, healthy ...

ANA, SMA, or anti-LKM1 ≥ 1:80 in adults and ≥ 1:20 in children; no AMA Hypergammaglobulinemia of any degree ANA, SMA or anti-LKM1 ≥ 1:40 in adults; other autoantibodies Interface hepatitis ...