明显的面部无力、上睑下垂和下颌下垂:一个与 RYR1 相关的先天性中心核肌病病例

IF 0.4 Q4 PEDIATRICS Journal of pediatric genetics Pub Date : 2021-07-03 eCollection Date: 2023-12-01 DOI:10.1055/s-0041-1731683
Bhanudeep Singanamalla, Shivan Kesavan, Divya Aggarwal, Debajyoti Chatterjee, Andoni Urtizberea, Renu Suthar
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引用次数: 0

摘要

先天性肌病是一种范围不断扩大的神经肌肉疾病,在婴儿期或儿童期发病,表现为肌张力低下和缓慢或非进行性骨骼肌无力。与 RYR1 相关的肌病是最常见和最常诊断的一类先天性肌病。恶性高热易感性和中枢核心病是常染色体显性或新发的 RYR1 疾病,而多核心病、先天性纤维型比例失调和中心核肌病则是常染色体隐性的 RYR1 疾病。上睑下垂、眼瘫、面部和近端肌肉无力,以及肌肉活检中出现尘核和多内核是诊断的线索。我们描述了一名 18 岁的男性患者,他在婴儿期就出现了上睑下垂、眼肌麻痹、肌病面容、下颌下垂和近端肌无力,经基因分析和肌肉活检确诊为与 RYR1 相关的先天性中心核肌病。
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Marked Facial Weakness, Ptosis, and Hanging Jaw: A Case with RYR1 -Related Congenital Centronuclear Myopathy.

Congenital myopathies are an expanding spectrum of neuromuscular disorders with early infantile or childhood onset hypotonia and slowly or nonprogressive skeletal muscle weakness. RYR1 -related myopathies are the most common and frequently diagnosed class of congenital myopathies. Malignant hyperthermia susceptibility and central core disease are autosomal dominant or de novo RYR1 disorder, whereas multiminicore, congenital fiber type disproportion and centronuclear myopathy are autosomal recessive RYR1 disorders. The presence of ptosis, ophthalmoparesis, facial, and proximal muscles weakness, with the presence of dusty cores and multiple internal nuclei on muscle biopsy are clues to the diagnosis. We describe an 18-year-old male, who presented with early infantile onset ptosis, ophthalmoplegia, myopathic facies, hanging lower jaw, and proximal muscle weakness confirmed as an RYR1 -related congenital centronuclear myopathy on genetic analysis and muscle biopsy.

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期刊介绍: The Journal of Pediatric Genetics is an English multidisciplinary peer-reviewed international journal publishing articles on all aspects of genetics in childhood and of the genetics of experimental models. These topics include clinical genetics, molecular genetics, biochemical genetics, medical genetics, dysmorphology, teratology, genetic counselling, genetic engineering, formal genetics, neuropsychiatric genetics, behavioral genetics, community genetics, cytogenetics, hereditary or syndromic cancer genetics, genetic mapping, reproductive genetics, fetal pathology and prenatal diagnosis, multiple congenital anomaly syndromes, and molecular embryology of birth defects. Journal of Pediatric Genetics provides an in-depth update on new subjects and current comprehensive coverage of the latest techniques used in the diagnosis of childhood genetics. Journal of Pediatric Genetics encourages submissions from all authors throughout the world. The following articles will be considered for publication: editorials, original and review articles, short report, rapid communications, case reports, letters to the editor, and book reviews. The aim of the journal is to share and disseminate knowledge between all disciplines in the field of pediatric genetics. This journal is a publication of the World Pediatric Society: http://www.worldpediatricsociety.org/ The Journal of Pediatric Genetics is available in print and online. Articles published ahead of print are available via the eFirst service on the Thieme E-Journals platform.
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