Surillo-Dahdah Laura, Morfi-Pagán Carlos A
Department of Neurology, Institute of Neuroscience, Manatí Medical Center, Manatí, PRI.
Department of Medical Education, Ponce Health Sciences University, Ponce, PRI.
Cureus. 2023 Sep 15;15(9):e45289. doi: 10.7759/cureus.45289. eCollection 2023 Sep.
This case series provides a diagnosis of myoclonus-dystonia syndrome (MDS) in two patients whose original presentation was thought to be Tourette's syndrome. The first patient presented with dystonia and myoclonus, which progressively worsened with age, and was diagnosed with an epsilon-sarcoglycan gene (SGCE) mutation. The patient's father, who was diagnosed in his childhood with Tourette's syndrome, also received genetic testing, which proved that to be a misdiagnosis and confirmed that he was the carrier of the SGCE mutation. Both patients were subjected to a levodopa trial, which proved to be an effective treatment. To our knowledge, these are the first reported cases of heterozygous pathogenic mutation of SGCE in Puerto Rico.
本病例系列报告了两名最初被诊断为妥瑞氏综合征的患者,最终确诊为肌阵挛性肌张力障碍综合征(MDS)。首例患者表现为肌张力障碍和肌阵挛,且症状随年龄增长逐渐加重,经诊断携带ε-肌聚糖基因(SGCE)突变。该患者的父亲在童年时被诊断为妥瑞氏综合征,此次也接受了基因检测,结果证实此前诊断有误,他也是SGCE突变的携带者。两名患者均接受了左旋多巴试验,结果证明该治疗有效。据我们所知,这是波多黎各首次报告的SGCE杂合致病性突变病例。