Debnath Anindya, Athani Badarinath, Das Jaya, Chandini N C
Physical Medicine and Rehabilitation, St. John's National Academy of Health Sciences, Bengaluru, IND.
Cureus. 2024 Nov 30;16(11):e74805. doi: 10.7759/cureus.74805. eCollection 2024 Nov.
Emery-Dreifuss Muscular Dystrophy (EDMD) is a rare genetic disorder characterized by muscle weakness, joint contractures, and cardiac dysfunction. Within this spectrum, EDMD Type 2, attributed to a heterozygous missense variant in exon 9 of the LMNA gene, presents a distinctive clinical profile. This case report details the presentation and management of a teenage girl displaying neck, trunk, upper and lower limb weakness, Achilles tendon contracture, and lordosis. Initial assessments, including elevated creatine phosphokinase (CPK) levels and electromyography (EMG) suggestive of myopathy, prompted further investigation. Genomic analysis using targeted gene sequencing using the ExomeDepth method subsequently confirmed the rare autosomal variant of EDMD Type 2 within the OMIM (Online Mendelian Inheritance in Man) phenotype. Given the heterogeneity of EDMD, cardiac evaluation becomes paramount in understanding its multifaceted nature. In this case, the patient underwent surgical correction, specifically, Achilles tendon release, and subsequent post-surgical rehabilitation. Notably, there was a marked improvement in ambulation, underscoring the significance of early release of contractures in enhancing overall functional outcomes. This comprehensive case study not only contributes insights into the clinical, genetic, and surgical aspects of EDMD Type 2 but also highlights the pivotal role of timely interventions in optimizing patient outcomes.
埃默里-德赖富斯肌营养不良症(EDMD)是一种罕见的遗传性疾病,其特征为肌肉无力、关节挛缩和心脏功能障碍。在这一疾病范围内,2型EDMD由LMNA基因第9外显子的杂合错义变异引起,具有独特的临床特征。本病例报告详细介绍了一名青少年女性的临床表现及治疗情况,该患者出现颈部、躯干、上肢和下肢无力、跟腱挛缩和脊柱前凸。包括肌酸磷酸激酶(CPK)水平升高和提示肌病的肌电图(EMG)在内的初步评估促使进行进一步检查。随后,使用ExomeDepth方法进行靶向基因测序的基因组分析证实了该患者存在OMIM(《人类孟德尔遗传》)表型中的2型EDMD罕见常染色体变异。鉴于EDMD的异质性,心脏评估对于理解其多方面性质至关重要。在本病例中,患者接受了手术矫正,具体为跟腱松解术,以及术后康复治疗。值得注意的是,患者的行走能力有显著改善,这突出了早期松解挛缩对于提高整体功能预后的重要性。这一全面的病例研究不仅有助于深入了解2型EDMD的临床、遗传和手术方面,还强调了及时干预在优化患者预后方面的关键作用。