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当呼吸成为一项挑战:一名患有DOK-7基因突变的印度新生儿先天性重症肌无力病例

When Breathing Becomes a Challenge: A Case of Congenital Myasthenia Gravis in an Indian Neonate With a DOK-7 Gene Mutation.

作者信息

Mendpara Vaidehi, Bethanabotla Sanjay, Yadav Megha, Kanisetti Vaishnavi, Singh Gurpreet, Das Abhirami, Sahu Sweta, Patel Hitesh

机构信息

Medicine and Surgery, Pediatrics, Government Medical College Surat, Surat, IND.

Internal Medicine, Osmania Medical College, Hyderabad, IND.

出版信息

Cureus. 2023 May 10;15(5):e38842. doi: 10.7759/cureus.38842. eCollection 2023 May.

Abstract

A rare neuromuscular condition known as congenital myasthenia gravis (CMG) affects some people from birth or very soon after. It results in fatigue and muscle weakness because of genetic abnormalities that interfere with the neuromuscular junction's ability to function, where the nerves and muscles connect. Even among those who have the same genetic mutation, the severity of CMG symptoms might differ considerably. The most typical signs of CMG include eyelid drooping, breathing issues, muscle weakness and weariness, and difficulties swallowing. Clinical examinations, neurophysiologic tests, and genetic analyses are frequently combined to make the diagnosis of CMG. Although there is no known treatment for CMG, many patients may control their symptoms and lead relatively normal lives with the right care. A newborn with CMG due to a DOK-7 gene mutation is described in this article, along with its very early onset. The DOK-7 mutation is a rare variant in the Indian population that causes CMG and usually manifests as 'limb girdle' weakness. However, due to muscle weakness, the neonate in this case developed severe respiratory distress and later died despite rigorous life-saving measures.

摘要

一种罕见的神经肌肉疾病,称为先天性重症肌无力(CMG),会影响一些人从出生时或出生后不久就开始发病。由于基因异常干扰了神经和肌肉连接部位(神经肌肉接头)的功能能力,从而导致疲劳和肌肉无力。即使在那些具有相同基因突变的人中,CMG症状的严重程度也可能有很大差异。CMG最典型的症状包括眼睑下垂、呼吸问题、肌肉无力和疲劳以及吞咽困难。临床检查、神经生理学测试和基因分析常常结合起来用于CMG的诊断。虽然目前尚无已知的CMG治疗方法,但许多患者通过适当的护理可以控制症状并过上相对正常的生活。本文描述了一名因DOK - 7基因突变而患有CMG的新生儿及其非常早的发病情况。DOK - 7突变在印度人群中是一种罕见的变异,会导致CMG,通常表现为“肢带型”肌无力。然而,由于肌肉无力,该病例中的新生儿尽管采取了严格的救生措施,仍出现了严重的呼吸窘迫并最终死亡。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/8985/10256249/48eb38bff738/cureus-0015-00000038842-i01.jpg

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