Grünewald Stephanie
Metabolic Medicine Unit, Great Ormond Street Hospital for Children NHS Trust with the UCL Institute of Child Health, London WC1N 3JH, UK.
Biochim Biophys Acta. 2009 Sep;1792(9):827-34. doi: 10.1016/j.bbadis.2009.01.003. Epub 2009 Jan 14.
Congenital disorders of glycosylation are a clinically and genetically heterogeneous group of disorders resulting from abnormal glycosylation of various glycoconjugates. The first description of congenital disorders of glycosylation was published in the early 80s and once screening tests for glycosylation disorders (CDGs) became readily available, CDG-Ia became the most frequently diagnosed CDG subtype. CDG-Ia is pan-ethnic and the spectrum of the clinical manifestations is still evolving: it spans from severe hydrops fetalis and fetal loss to a (nearly) normal phenotype. However, the most common presentation in infancy is of a multisystem disorder with central nervous system involvement.
糖基化先天性疾病是一组临床和遗传异质性疾病,由各种糖缀合物的异常糖基化引起。糖基化先天性疾病的首次描述发表于80年代初,一旦糖基化疾病(CDG)的筛查测试变得容易获得,CDG-Ia就成为最常被诊断出的CDG亚型。CDG-Ia具有全种族性,其临床表现谱仍在不断演变:从严重的胎儿水肿和胎儿丢失到(几乎)正常的表型。然而,婴儿期最常见的表现是伴有中枢神经系统受累的多系统疾病。