Lashkari A, Smith A K, Graham J M
Steven Spielberg Pediatric Research Center, Ahmanson Pediatric Center, UCLA School of Medicine.
Clin Pediatr (Phila). 1999 Apr;38(4):189-208. doi: 10.1177/000992289903800401.
Williams-Beuren syndrome is an autosomal dominant disorder resulting from a submicroscopic deletion of contiguous genes on the long arm of chromosome 7. It consists of a variety of hallmark physical features, which include distinctive facial characteristics, cardiac anomalies (of which the most common is supravalvular aortic stenosis), and occasional idiopathic hypercalcemia. The condition also includes a unique cognitive profile, with relative sparing of language and facial recognition skills against a background of mental retardation. This paper reviews the early history and clinical experience with this syndrome, how it unfolds from infancy through adulthood, and how it manifests in different organ systems. Evidence-based recommendations are then offered for the treatment of the specific developmental and medical issues that arise in patients with Williams syndrome.
威廉姆斯-贝伦综合征是一种常染色体显性疾病,由7号染色体长臂上相邻基因的亚显微缺失引起。它具有多种标志性的身体特征,包括独特的面部特征、心脏异常(其中最常见的是瓣膜上主动脉狭窄)以及偶尔出现的特发性高钙血症。这种疾病还具有独特的认知特征,在智力发育迟缓的背景下,语言和面部识别技能相对保留。本文回顾了该综合征的早期历史和临床经验,其从婴儿期到成年期的发展过程,以及在不同器官系统中的表现。然后针对威廉姆斯综合征患者出现的特定发育和医学问题的治疗提供基于证据的建议。