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Transatlantic combined and comparative data analysis of 1095 patients with urea cycle disorders—A successful strategy for clinical research of rare diseases

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To improve our understanding of urea cycle disorders (UCDs) prospectively followed by two North American (NA) and European (EU) patient cohorts. Click to show full abstract

To improve our understanding of urea cycle disorders (UCDs) prospectively followed by two North American (NA) and European (EU) patient cohorts.

Keywords: combined comparative; comparative data; cycle disorders; urea cycle; transatlantic combined

Journal Title: Journal of Inherited Metabolic Disease
Year Published: 2019

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