Outcome of the glutaric aciduria type 1 (GA1) newborn screening program in Manitoba: 1980-2020

曼尼托巴省戊二酸尿症1型(GA1)新生儿筛查项目的结果:1980-2020年

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Abstract

Glutaric aciduria type 1 (GA1) is a severe inherited neurometabolic disorder whose clinical outcome has improved after implementation of newborn screening (NBS) programs and prompt beginning of guideline-directed presymptomatic metabolic treatment. We report the outcome of our 40-year experience with the diagnosis and management of GA1 which has improved but remains suboptimal.

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