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一项导致脊髓性肌萎缩症延迟诊断的深刻观察:病例报告

An Insightful Observation Leading to a Late Diagnosis of Spinal Muscular Atrophy: A Case Report.

作者信息

Rivera Troia Felix, Ocasio Villa Fernando

机构信息

Genetics, University of Medicine and Health Sciences, Basseterre, KNA.

Genetics, Western Oncology Cancer Center, Mayagüez Medical Center, Mayagüez, PRI.

出版信息

Cureus. 2024 May 7;16(5):e59786. doi: 10.7759/cureus.59786. eCollection 2024 May.

Abstract

Spinal muscular atrophy (SMA) is a rare autosomal recessive neuromuscular disorder characterized by the loss of motor neurons in the spinal cord that results in progressive muscle weakness and atrophy. Most often, the gene involved in this disorder is the survival motor neuron (SMN1) gene, located on the telomeric regions of chromosome 5q13. This gene is involved in the processing of pre-mRNA required for the formation of dendrites and axons. Here we present the case of a 47-year-old female with an extensive past medical history of progressive muscle weakness who, after numerous specialist evaluations, was sent for germline mutation panel sequencing and analysis and was incidentally found to have a pathogenic heterozygous deletion encompassing the exon 8 region of the SMN1 gene. This case report aims to highlight the importance of timely identification and management for individuals who present with early clinical signs of the disease to reduce the morbidity and mortality associated with it.

摘要

脊髓性肌萎缩症(SMA)是一种罕见的常染色体隐性神经肌肉疾病,其特征是脊髓中的运动神经元丧失,导致进行性肌肉无力和萎缩。最常见的是,参与这种疾病的基因是生存运动神经元(SMN1)基因,位于5号染色体q13的端粒区域。该基因参与树突和轴突形成所需的前体mRNA的加工。在此,我们报告一例47岁女性病例,该患者有广泛的进行性肌肉无力病史,经过多次专科评估后,被送去进行种系突变检测板测序和分析,偶然发现其存在一个致病性杂合缺失,涵盖SMN1基因的外显子8区域。本病例报告旨在强调及时识别和管理出现该疾病早期临床症状个体的重要性,以降低与之相关的发病率和死亡率。

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RNA in spinal muscular atrophy: therapeutic implications of targeting.脊髓性肌萎缩症中的 RNA:靶向治疗的意义。
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