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Gene Therapy
Gene Therapy for Ornithine Transcarbamylase Deficiency
Recruiting1 awardPhase 3
Little Rock, Arkansas
This trial is testing DTX301, a treatment designed to help people with late-onset OTC deficiency, a condition that makes it hard for their bodies to process certain proteins. The treatment aims to improve the function of an enzyme that helps manage ammonia levels in the blood. Participants will be monitored for several years, with some continuing in a follow-up program for additional time.
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