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Urea Cycle Disorders
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Urea Cycle Disorders trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
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Ornithine Transcarbamylase (OTC) deficiency, the most common urea cycle disorder, is an inherited metabolic disorder caused by a genetic defect in a liver enzym…
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…
This study is being done to understand how the brain works while people with urea cycle disorder (UCD) perform driving tasks that range from easy to difficult a…
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…
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approa…
The primary objective is to evaluate the efficacy of DTX301 on the improvement of ornithine transcarbamylase (OTC) function by maintaining safe plasma ammonia l…