Claudia Dosi, Anna Rubegni, Denise Cassandrini, Alessandro Malandrini, Lorenzo Maggi, M Alice Donati, Filippo M Santorelli
{"title":"α -肌糖病表现为肌痛和高血凝症在两个成人长期随访。案例报告。","authors":"Claudia Dosi, Anna Rubegni, Denise Cassandrini, Alessandro Malandrini, Lorenzo Maggi, M Alice Donati, Filippo M Santorelli","doi":"10.36185/2532-1900-025","DOIUrl":null,"url":null,"abstract":"<p><p>Two patients with a paucisymptomatic hyperckemia underwent a skeletal muscle biopsy and massive gene panel to investigate mutations associated with inherited muscle disorders. In the SGCA gene, sequence analyses revealed a homozygous c.850C > T/p.Arg284Cys in patient 1 and two heterozygous variants (c.739G > A/p.Val247Met and c.850C > T/p.Arg284Cys) in patient 2. Combination of histology and immunofluorence studies showed minimal changes for muscular proteins including the α-sarcoglycan. These two cases highlight the advantages of next-generation sequencing in the differential diagnosis of mild myopathic conditions before considering the more invasive muscle biopsy in sarcoglycanopathies.</p>","PeriodicalId":35953,"journal":{"name":"Acta Myologica","volume":"39 4","pages":"218-221"},"PeriodicalIF":0.0000,"publicationDate":"2020-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/64/8b/am-2020-04-218.PMC7783442.pdf","citationCount":"1","resultStr":"{\"title\":\"Alpha-sarcoglycanopathy presenting as myalgia and hyperCKemia in two adults with a long-term follow-up. Case reports.\",\"authors\":\"Claudia Dosi, Anna Rubegni, Denise Cassandrini, Alessandro Malandrini, Lorenzo Maggi, M Alice Donati, Filippo M Santorelli\",\"doi\":\"10.36185/2532-1900-025\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Two patients with a paucisymptomatic hyperckemia underwent a skeletal muscle biopsy and massive gene panel to investigate mutations associated with inherited muscle disorders. In the SGCA gene, sequence analyses revealed a homozygous c.850C > T/p.Arg284Cys in patient 1 and two heterozygous variants (c.739G > A/p.Val247Met and c.850C > T/p.Arg284Cys) in patient 2. Combination of histology and immunofluorence studies showed minimal changes for muscular proteins including the α-sarcoglycan. These two cases highlight the advantages of next-generation sequencing in the differential diagnosis of mild myopathic conditions before considering the more invasive muscle biopsy in sarcoglycanopathies.</p>\",\"PeriodicalId\":35953,\"journal\":{\"name\":\"Acta Myologica\",\"volume\":\"39 4\",\"pages\":\"218-221\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2020-12-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/64/8b/am-2020-04-218.PMC7783442.pdf\",\"citationCount\":\"1\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Acta Myologica\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.36185/2532-1900-025\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q3\",\"JCRName\":\"Medicine\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Acta Myologica","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.36185/2532-1900-025","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"Medicine","Score":null,"Total":0}
Alpha-sarcoglycanopathy presenting as myalgia and hyperCKemia in two adults with a long-term follow-up. Case reports.
Two patients with a paucisymptomatic hyperckemia underwent a skeletal muscle biopsy and massive gene panel to investigate mutations associated with inherited muscle disorders. In the SGCA gene, sequence analyses revealed a homozygous c.850C > T/p.Arg284Cys in patient 1 and two heterozygous variants (c.739G > A/p.Val247Met and c.850C > T/p.Arg284Cys) in patient 2. Combination of histology and immunofluorence studies showed minimal changes for muscular proteins including the α-sarcoglycan. These two cases highlight the advantages of next-generation sequencing in the differential diagnosis of mild myopathic conditions before considering the more invasive muscle biopsy in sarcoglycanopathies.