Transatlantic combined and comparative data analysis of 1095 patients with urea cycle disorders-A successful strategy for clinical research of rare diseases. [electronic resource]
- Journal of inherited metabolic disease 01 2019
- 93-106 p. digital
Publication Type: Comparative Study; Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
1573-2665
10.1002/jimd.12031 doi
Cohort Studies Data Analysis Delayed Diagnosis Europe Female Humans Infant, Newborn Male Neonatal Screening--methods North America Ornithine Carbamoyltransferase Deficiency Disease--diagnosis Rare Diseases Urea--metabolism Urea Cycle Disorders, Inborn--diagnosis