{"title":"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","authors":"Bhanudeep Singanamalla, Shivan Kesavan, Divya Aggarwal, Debajyoti Chatterjee, Andoni Urtizberea, Renu Suthar","doi":"10.1055/s-0041-1731683","DOIUrl":null,"url":null,"abstract":"<p><p>Congenital myopathies are an expanding spectrum of neuromuscular disorders with early infantile or childhood onset hypotonia and slowly or nonprogressive skeletal muscle weakness. <i>RYR1</i> -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 <i>RYR1</i> disorder, whereas multiminicore, congenital fiber type disproportion and centronuclear myopathy are autosomal recessive <i>RYR1</i> 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 <i>RYR1</i> -related congenital centronuclear myopathy on genetic analysis and muscle biopsy.</p>","PeriodicalId":55037,"journal":{"name":"IAWA Journal","volume":"10 1","pages":"318-324"},"PeriodicalIF":1.4000,"publicationDate":"2021-07-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10756716/pdf/","citationCount":"0","resultStr":"{\"title\":\"Marked Facial Weakness, Ptosis, and Hanging Jaw: A Case with <i>RYR1</i> -Related Congenital Centronuclear Myopathy.\",\"authors\":\"Bhanudeep Singanamalla, Shivan Kesavan, Divya Aggarwal, Debajyoti Chatterjee, Andoni Urtizberea, Renu Suthar\",\"doi\":\"10.1055/s-0041-1731683\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Congenital myopathies are an expanding spectrum of neuromuscular disorders with early infantile or childhood onset hypotonia and slowly or nonprogressive skeletal muscle weakness. <i>RYR1</i> -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 <i>RYR1</i> disorder, whereas multiminicore, congenital fiber type disproportion and centronuclear myopathy are autosomal recessive <i>RYR1</i> 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 <i>RYR1</i> -related congenital centronuclear myopathy on genetic analysis and muscle biopsy.</p>\",\"PeriodicalId\":55037,\"journal\":{\"name\":\"IAWA Journal\",\"volume\":\"10 1\",\"pages\":\"318-324\"},\"PeriodicalIF\":1.4000,\"publicationDate\":\"2021-07-03\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10756716/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"IAWA Journal\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1055/s-0041-1731683\",\"RegionNum\":3,\"RegionCategory\":\"农林科学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2023/12/1 0:00:00\",\"PubModel\":\"eCollection\",\"JCR\":\"Q2\",\"JCRName\":\"FORESTRY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"IAWA Journal","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1055/s-0041-1731683","RegionNum":3,"RegionCategory":"农林科学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2023/12/1 0:00:00","PubModel":"eCollection","JCR":"Q2","JCRName":"FORESTRY","Score":null,"Total":0}
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.
期刊介绍:
The IAWA Journal is the only international periodical fully devoted to structure, function, identification and utilisation of wood and bark in trees, shrubs, lianas, palms, bamboo and herbs. Many papers are of a multidisciplinary nature, linking