ORNITHINE TRANSCARBAMYLASE (OTC) DEFICIENCY

Clinical trials for ORNITHINE TRANSCARBAMYLASE (OTC) DEFICIENCY explained in plain language.

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Scientists track Long-Term safety of One-Time gene fix for rare disorder

Disease control Ongoing

This study is tracking the long-term safety and effectiveness of a one-time gene therapy for adults with late-onset OTC deficiency, a rare genetic disorder that affects the body's ability to process protein. It follows 11 adults who previously received the experimental treatment …

Matched conditions: ORNITHINE TRANSCARBAMYLASE (OTC) DEFICIENCY

Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control

Last updated Mar 24, 2026 12:02 UTC

ORNITHINE TRANSCARBAMYLASE (OTC) DEFICIENCY

Get alerted when new ORNITHINE TRANSCARBAMYLASE (OTC) DEFICIENCY trials appear

Scientists track Long-Term safety of One-Time gene fix for rare disorder