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.
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http://dx.doi.org/10.7759/cureus.74805 | DOI Listing |
Cureus
November 2024
Physical Medicine and Rehabilitation, St. John's National Academy of Health Sciences, Bengaluru, IND.
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.
View Article and Find Full Text PDFCells
January 2024
CNR Institute of Molecular Genetics "Luigi Luca Cavalli-Sforza", Unit of Bologna, 40136 Bologna, Italy.
In muscle cells subjected to mechanical stimulation, LINC complex and cytoskeletal proteins are basic to preserve cellular architecture and maintain nuclei orientation and positioning. In this context, the role of lamin A/C remains mostly elusive. This study demonstrates that in human myoblasts subjected to mechanical stretching, lamin A/C recruits desmin and plectin to the nuclear periphery, allowing a proper spatial orientation of the nuclei.
View Article and Find Full Text PDFJ Physiol Sci
November 2023
Department of Pathophysiology, Tokyo Medical University, Tokyo, Japan.
Emery-Dreifuss muscular dystrophy (EDMD), caused by mutations in genes encoding nuclear envelope proteins, is clinically characterized by muscular dystrophy, early joint contracture, and life-threatening cardiac abnormalities. To elucidate the pathophysiological mechanisms underlying striated muscle involvement in EDMD, we previously established a murine model with mutations in Emd and Lmna (Emd/Lmna; EH), and reported exacerbated skeletal muscle phenotypes and no notable cardiac phenotypes at 12 weeks of age. We predicted that lack of emerin in Lmna mice causes an earlier onset and more pronounced cardiac dysfunction at later stages.
View Article and Find Full Text PDFEur Heart J
December 2023
Institute of Cardiovascular Science, University College London, London, UK.
Background And Aims: Emery-Dreifuss muscular dystrophy (EDMD) is caused by variants in EMD (EDMD1) and LMNA (EDMD2). Cardiac conduction defects and atrial arrhythmia are common to both, but LMNA variants also cause end-stage heart failure (ESHF) and malignant ventricular arrhythmia (MVA). This study aimed to better characterize the cardiac complications of EMD variants.
View Article and Find Full Text PDFJ Clin Invest
July 2023
Department of Molecular Biology.
Mutations in genes encoding nuclear envelope proteins lead to diseases known as nuclear envelopathies, characterized by skeletal muscle and heart abnormalities, such as Emery-Dreifuss muscular dystrophy (EDMD). The tissue-specific role of the nuclear envelope in the etiology of these diseases has not been extensively explored. We previously showed that global deletion of the muscle-specific nuclear envelope protein NET39 in mice leads to neonatal lethality due to skeletal muscle dysfunction.
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