Yeditepe University Department of Pediatrics, İstanbul, Turkey.
Acta Myol. 2020 Dec 1;39(4):266-273. doi: 10.36185/2532-1900-029. eCollection 2020 Dec.
Congenital myopathies represent a clinically and genetically heterogeneous group of early-onset neuromuscular diseases with characteristic, but not always specific, histopathological features, often presenting with stable and/or slowly progressive truncal and proximal weakness. It is often not possible to have a diagnosis on clinical ground alone. Additional extraocular, respiratory, distal involvement, scoliosis, and distal laxity may provide clues. The "core myopathies" collectively represent the most common form of congenital myopathies, and the name pathologically corresponds to histochemical appearance of focally reduced oxidative enzyme activity and myofibrillar changes on ultrastructural studies. Because of the clinical, pathological, and molecular overlaps, central core disease and multiminicore disease will be discussed together.
先天性肌病是一组具有特征性但并非总是特异性的组织病理学特征的早发性神经肌肉疾病,临床上和基因上具有异质性,常表现为稳定和/或缓慢进行性的躯干和近端肌无力。仅凭临床检查通常无法做出诊断。额外的眼外肌、呼吸、远端受累、脊柱侧凸和远端松弛可能提供线索。“核心性肌病”共同代表了最常见的先天性肌病形式,其病理名称与组织化学上局灶性氧化酶活性降低和超微结构研究中肌原纤维变化的外观相对应。由于临床、病理和分子上的重叠,中央核心病和多发性核心病将一起讨论。