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Urea Cycle Disorders, Inborn
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Urea Cycle Disorders, Inborn trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
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The goal of this observational study is to learn if people with certain ammonia metabolism disorders will measure their ammonia levels at home. The main questi…
Patients with high blood pressure (hypertension) and chronic kidney disease are at an increased risk of developing heart disease and strokes. Part of this risk…
Arginase 1 deficiency (ARG1-D) is a rare condition in which the body cannot properly break down a substance called arginine. This leads to high levels of argini…
Urea cycle disorders (UCD) are rare diseases in China, would lead to high mortality and disability, which require long-term management due to the recurrent symp…
Ornithine Transcarbamylase (OTC) deficiency, the most common urea cycle disorder, is an inherited metabolic disorder caused by a genetic defect in a liver enzym…
Urea cycle disorders (UCDs) are dramatic congenital inherited metabolic disorders. There is no cure. Many novel therapeutic approaches are currently being devel…
This is a single-site Phase 1/2 open-label umbrella clinical trial designed to evaluate the safety, tolerability, and efficacy of a single intravenous dose of L…
The primary objective is to evaluate the efficacy of DTX301 on the improvement of ornithine transcarbamylase (OTC) function by maintaining safe plasma ammonia l…