OTC DEFICIENCY
Clinical trials for OTC DEFICIENCY explained in plain language.
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New injection aims to control rare genetic liver disease
Disease control OngoingThis early-stage study tests a new drug, CMP-CPS-001, given as a shot under the skin. It includes 120 healthy adults and people with a genetic condition called OTC deficiency, which affects the liver's ability to remove ammonia from the body. The main goal is to check safety and …
Matched conditions: OTC DEFICIENCY
Phase: PHASE1 • Sponsor: CAMP4 Therapeutics Corporation • Aim: Disease control
Last updated May 26, 2026 10:25 UTC
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Gene therapy shows promise for rare metabolic disorder
Disease control OngoingThis study tests a gene therapy called DTX301 for adults with late-onset OTC deficiency, a genetic disorder that causes dangerous ammonia buildup in the blood. The goal is to see if a single treatment can safely keep ammonia levels under control and reduce the need for emergency …
Matched conditions: OTC DEFICIENCY
Phase: PHASE3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated May 26, 2026 10:22 UTC