The objective of this study was to compare survival rate, neurocognitive function, and quality of life in patients with neonatal onset urea cycle disorders (UCDs) who received liver transplants versus those managed medically.

Project page: Comparing Treatment Options for Urea Cycle Disorders
Principal Investigator: Nicholas Ah Mew, MD
Organization: Children's Research Institute
Original project title: Comparative Effectiveness of Therapy in Rare Diseases: Liver Transplantation vs. Conservative Management of Urea Cycle Disorders
HSRProj ID: 20162105 ID: NCT02740153

To cite this document, please use: Ah Mew N, McCarter R, Izem R, et al. (2020). Comparing Treatment Options for Urea Cycle Disorders. Patient-Centered Outcomes Research Institute (PCORI).


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