Rossi Massimiliano, Jones Rachel L, Norbury Gail, Bloch-Zupan Agnès, Winter Robin M
Clinical & Molecular Genetics Unit, Level 5 Camelia Botnar Labs, Great Ormond Street Hospital for Children NHS Trust, London, UK.
Clin Dysmorphol. 2003 Oct;12(4):269-74. doi: 10.1097/00019605-200310000-00012.
Patients affected by Pfeiffer syndrome generally present with syndromic craniosynostosis and typical limb defects including broad thumbs, wide halluces with varus deformity, toe syndactyly and sometimes elbow ankylosis. This autosomal dominant condition can be caused by mutations in either fibroblast growth factor receptor gene type 1 or 2 (FGFR1 or FGFR2). We report four new affected families showing an FGFR1 P252R mutation and emphasize the characteristic malformations of the feet in this form of Pfeiffer syndrome. In one family this was the only abnormality.
患有 Pfeiffer 综合征的患者通常表现为综合征性颅缝早闭和典型的肢体缺陷,包括拇指宽大、拇趾宽且有内翻畸形、足趾并指,有时还有肘关节强直。这种常染色体显性疾病可由 1 型或 2 型成纤维细胞生长因子受体基因(FGFR1 或 FGFR2)突变引起。我们报告了四个新的患病家族,其显示出 FGFR1 P252R 突变,并强调了这种形式的 Pfeiffer 综合征中足部的特征性畸形。在一个家族中,这是唯一的异常情况。