Transatlantic combined and comparative data analysis of 1095 patients with urea cycle disorders—a successful strategy for clinical research of rare diseases
Journal of Inherited Metabolic Disease(2019)
摘要
Combined analysis of databases drawn from distinct populations opens the possibility to increase sample sizes for natural history questions, while comparative analysis utilising differences in approach to treatment can evaluate therapeutic options and enhance long-term outcome studies.
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关键词
Urea cycle Disorders,diagnostic methods,international registry and database
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