成人发病远端肌病与主要手累及作为一个罕见的表型的胸电病。

IF 3.2 4区 医学 Q2 CLINICAL NEUROLOGY
Laura Llansó, David Reyes-Leiva, Alba Segarra-Casas, Tomàs Xuclà-Ferrarons, Eduard Gallardo, Rosa Blanco, Pia Gallano, Montse Olivé, Lidia González-Quereda
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引用次数: 0

摘要

典型的凝集菌病表型包括单纯大疱性表皮松解症和近端分布的肌肉萎缩症,伴有或不伴有皮肤起泡。然而,近年来,患者表现出新的肌肉表型,包括下肢远端无力已被描述。我们的目的是扩大表型谱的胸纤腺病报告的情况下,表现为纯粹的骨骼肌病主要上肢远端无力。我们描述这种远端肌病表型提供全面的临床,遗传,肌病理和放射学数据。遗传学研究确定了PLEC的两个截断变异体。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Adult-onset distal myopathy with predominant hand involvement as a rare phenotype of plectinopathy.

Classic phenotypes of plectinopathies include epidermolysis bullosa simplex and muscular dystrophy with proximal distribution, associated or not with skin blistering. However, in recent years, patients manifesting new muscular phenotypes including lower-limb distal weakness have been described. We aim to expand the phenotypic spectrum of plectinopathies by reporting a case presenting with a pure skeletal myopathy with predominant upper-limb distal weakness. We describe this distal myopathy phenotype providing comprehensive clinical, genetic, myopathological and radiological data. Genetic studies identified two truncating variants in PLEC.

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来源期刊
Journal of neuromuscular diseases
Journal of neuromuscular diseases Medicine-Neurology (clinical)
CiteScore
5.10
自引率
6.10%
发文量
102
期刊介绍: The Journal of Neuromuscular Diseases aims to facilitate progress in understanding the molecular genetics/correlates, pathogenesis, pharmacology, diagnosis and treatment of acquired and genetic neuromuscular diseases (including muscular dystrophy, myasthenia gravis, spinal muscular atrophy, neuropathies, myopathies, myotonias and myositis). The journal publishes research reports, reviews, short communications, letters-to-the-editor, and will consider research that has negative findings. The journal is dedicated to providing an open forum for original research in basic science, translational and clinical research that will improve our fundamental understanding and lead to effective treatments of neuromuscular diseases.
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