Bushby K, Anderson L V, Pollitt C, Naom I, Muntoni F, Bindoff L
Department of Human Genetics, University of Newcastle upon Tyne, UK.
Brain. 1998 Apr;121 ( Pt 4):581-8. doi: 10.1093/brain/121.4.581.
We have identified seven patients (including two sib pairs) with a predominantly late onset limb-girdle muscular dystrophy in whom an absence of merosin was noted on immunoblotting. Merosin immunocytochemistry was normal, and no abnormalities were detected on immunostaining for the various proteins known to be involved in the limb-girdle muscular dystrophies (alpha, beta, gamma, delta sarcoglycan and calpain 3). Apart from one patient, where muscle problems began in childhood, reported age at onset of muscle weakness involving initially the proximal muscles of the lower limbs ranged from 17 to 40 years. The pattern of muscle involvement was similar from patient to patient, with hypertrophy of at least the calf muscles, absence of scapular winging and predominant involvement of hip flexors and adductors and hamstrings more than quadriceps. Serum creatine kinase in all patients was at least 10 times normal, and muscle biopsies showed non-specific dystrophic features. We believe that the patients described here may represent a genetically distinct subset within the limb-girdle muscular dystrophy group.
我们已确定了7名患者(包括2对同胞兄弟姊妹),他们主要患有迟发性肢带型肌营养不良症,免疫印迹显示其缺乏merosin。Merosin免疫细胞化学结果正常,对已知与肢带型肌营养不良症相关的各种蛋白质(α、β、γ、δ肌聚糖和钙蛋白酶3)进行免疫染色时未检测到异常。除了一名患者肌肉问题始于儿童期外,报告的下肢近端肌肉最初出现肌无力的发病年龄范围为17至40岁。患者之间的肌肉受累模式相似,至少腓肠肌肥大,无肩胛翼状突起,髋部屈肌和内收肌以及绳肌比股四头肌受累更明显。所有患者的血清肌酸激酶至少是正常水平的10倍,肌肉活检显示非特异性营养不良特征。我们认为这里描述的患者可能代表肢带型肌营养不良症组内一个基因上不同的亚组。