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两名具有不同突变的新生儿的先天性杆状体肌病:病例系列及文献综述

Congenital Nemaline Myopathy in Two Neonates With Different Mutations: A Case Series and Literature Review.

作者信息

Mohtisham Farzeen, Al Thaqafi Maram, Shawli Aiman, Sallam Adel

机构信息

Neonatology, King Saud Bin Abdulaziz University for Health Sciences, Jeddah, SAU.

Neonatology, King Abdulaziz Medical City, Ministry of National Guard Health Affairs, Jeddah, SAU.

出版信息

Cureus. 2023 Sep 13;15(9):e45197. doi: 10.7759/cureus.45197. eCollection 2023 Sep.

Abstract

Nemaline myopathy is a skeletal muscle disorder characterized by a wide range of severity and variable presentation. While most cases present in the neonatal period with symptoms, such as hypotonia, muscle weakness, and respiratory insufficiency, delayed onset in childhood or adulthood is also observed. The pathogenesis of nemaline myopathy involves at least 12 genes, and the condition can arise from de novo mutations or be inherited in a dominant or recessive manner. In this study, we present two cases of neonates admitted to a neonatal intensive care unit (NICU) exhibiting hypotonia, muscle weakness, and respiratory insufficiency. Both cases were diagnosed with congenital nemaline myopathy, with each patient displaying distinct mutations. This report highlights the clinical and genetic heterogeneity of this condition, emphasizing the importance of early recognition and genetic evaluation for accurate diagnosis and appropriate management of affected individuals.

摘要

杆状体肌病是一种骨骼肌疾病,其严重程度范围广泛,表现多样。虽然大多数病例在新生儿期出现症状,如肌张力减退、肌肉无力和呼吸功能不全,但也观察到在儿童期或成年期发病较晚的情况。杆状体肌病的发病机制涉及至少12个基因,这种疾病可由新发突变引起,或以显性或隐性方式遗传。在本研究中,我们报告了两例入住新生儿重症监护病房(NICU)的新生儿,他们表现出肌张力减退、肌肉无力和呼吸功能不全。两例均被诊断为先天性杆状体肌病,每位患者都有不同的突变。本报告强调了这种疾病的临床和遗传异质性,强调了早期识别和基因评估对于准确诊断和妥善管理受影响个体的重要性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d16c/10503874/2246ddb5a22b/cureus-0015-00000045197-i01.jpg

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