肉碱
新生儿筛查
脑病
医学
呕吐
儿科
β氧化
胃肠病学
酰基辅酶A脱氢酶
内科学
疾病
脱氢酶
酶
新陈代谢
生物化学
生物
作者
Xiaohong Chen,Yun Sun,Yanling Yang,Lianshu Han,Xinwen Huang
出处
期刊:Chinese journal of medical genetics
[Sichuan University School of Medicine]
日期:2021-05-10
卷期号:38 (5): 414-418
标识
DOI:10.3760/cma.j.cn511374-20200829-00630
摘要
Multiple acyl-CoA dehydrogenase deficiency (MADD), also known as glutaricacidemia type II, is a relatively common disorder of fatty acid oxidation metabolism. The clinical manifestations are highly heterogeneous, symptoms can develop from newborn to adulthood. Neonatal onset type is more serious with high mortality. The symptoms of late onset patients include lipid deposition myopathy and vomiting, liver disease, and encephalopathy. Analysis of blood acyl carnitine spectrum by tandem mass spectrometry can be used for the screening. Late onset patients have relatively good prognosis with vitamin B2 treatment. The purpose of this consensus is to standardize the diagnosis, treatment and management of MADD, so as to improve the prognosis of patients and reduce death and disability.
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