4.5 Review

An overview of inborn errors of metabolism manifesting with primary adrenal insufficiency

期刊

出版社

SPRINGER
DOI: 10.1007/s11154-018-9447-2

关键词

Adrenal insufficiency; Congenital adrenal hyperplasia; 21-hydroxylase deficiency; X-ALD; Niemann-pick diseases; Genetics; Mitochondrial disease

资金

  1. Intramural Research Programs of the National Institutes of Health Clinical Center
  2. Eunice Kennedy Shriver National Institute of Child Health of Human Development (NICHD)

向作者/读者索取更多资源

Primary adrenal insufficiency (PAI) results from an inability to produce adequate amounts of steroid hormones from the adrenal cortex. The most common causes of PAI are autoimmune adrenalitis (Addison's disease), infectious diseases, adrenalectomy, neoplasia, medications, and various rare genetic syndromes and inborn errors of metabolism that typically present in childhood although late-onset presentations are becoming increasingly recognized. The prevalence of PAI in Western countries is approximately 140 cases per million, with an incidence of 4 per 1,000,000 per year. Several pitfalls in the genetic diagnosis of patients with PAI exist. In this review, we provide an in-depth discussion and overview on the inborn errors of metabolism manifesting with PAI, including genetic diagnosis, genotype-phenotype relationships and counseling of patients and their families with a focus on various enzymatic deficiencies of steroidogenesis.

作者

我是这篇论文的作者
点击您的名字以认领此论文并将其添加到您的个人资料中。

评论

主要评分

4.5
评分不足

次要评分

新颖性
-
重要性
-
科学严谨性
-
评价这篇论文

推荐

暂无数据
暂无数据