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宫下肌病的临床表现、诊断及遗传学见解:一例报告及文献综述

Clinical Presentation, Diagnosis, and Genetic Insights of Miyoshi Myopathy: A Case Report and Literature Review.

作者信息

Stolarski Łukasz, Patrzałek Patryk, Gerber Friederike, Tokarczyk Wojciech, Bialasik-Misiorny Maksymilian, Kulma Marek

机构信息

Intensive Care Unit, District Hospital in Rawicz, Rawicz, POL.

Surgery, District Hospital in Rawicz, Rawicz, POL.

出版信息

Cureus. 2024 Sep 7;16(9):e68869. doi: 10.7759/cureus.68869. eCollection 2024 Sep.

Abstract

Miyoshi myopathy (MM) is an autosomal recessive dysferlinopathy caused by a mutation in the dysferlin (DYSF) gene on chromosome 2p. Recent findings indicate that MM and Lower Girdle Muscular Dystrophy R2 (LGMD2B) are the same disease. We present the case of a 44-year-old male who first experienced symptoms of MM at the age of 19, initially noticing difficulty climbing stairs and standing on his toes. By the age of 29, he had developed significant calf muscle atrophy and weakness, which led to difficulties with walking. Electromyography and nerve conduction studies showed axonal damage and myogenic features. Genetic testing ruled out Charcot-Marie-Tooth disease but identified a pathogenic variant in the DYSF gene. Laboratory tests revealed elevated creatine kinase levels. Photographs of the patient's lower limbs showed significant calf muscle atrophy. Based on clinical, laboratory, and electrophysiological findings, he was diagnosed with MM. This case highlights the importance of genetic testing in diagnosing muscular dystrophies and underscores the need for continued research into gene and cell therapies. To the best of our knowledge, this is one of the first studies reporting a case of MM in Poland.

摘要

宫下肌病(MM)是一种常染色体隐性遗传性肌膜蛋白病,由2号染色体短臂上的肌膜蛋白(DYSF)基因突变引起。最近的研究结果表明,MM与下半身型肌营养不良症R2型(LGMD2B)是同一种疾病。我们报告了一例44岁男性病例,他19岁时首次出现MM症状,最初是爬楼梯和踮脚尖困难。到29岁时,他出现了明显的小腿肌肉萎缩和无力,导致行走困难。肌电图和神经传导研究显示有轴突损伤和肌源性特征。基因检测排除了夏科-马里-图斯病,但在DYSF基因中发现了一个致病变异。实验室检查显示肌酸激酶水平升高。患者下肢照片显示小腿肌肉明显萎缩。根据临床、实验室和电生理检查结果,他被诊断为MM。该病例突出了基因检测在诊断肌营养不良症中的重要性,并强调了对基因和细胞疗法持续研究的必要性。据我们所知,这是波兰首例报告的MM病例研究之一。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fb45/11457810/710672d85051/cureus-0016-00000068869-i01.jpg

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