Recent insights into limb-girdle muscular dystrophy: Impacts, therapy, and challenges
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Date
2025
Authors
Jiang-Ling Xiao
Zhe Zhao
Heng-Yuan Liu
Chang-Fa Tang
Chen-Chen Sun
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Journal ISSN
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Abstract
Limb-girdle muscular dystrophy (LGMD) is a genetically heterogeneous group of muscle disorders characterized by progressive muscle atrophy and loss of motor function. Over 30 subtypes have been identified and classified into two main inheritance patterns: autosomal dominant and autosomal recessive. Besides primarily affecting skeletal muscle, certain subtypes also impact the cardiac and respiratory muscles, significantly influencing disease progression and patient survival. Substantial progress has been made in understanding the pathogenic genes and molecular mechanisms of LGMD; however, developing disease-modifying therapies remains challenging due to genetic heterogeneity, limitations in gene delivery technologies, and secondary pathological complications. Current treatments are primarily supportive, focusing on symptom management and improving quality of life rather than addressing the underlying cause. This paper summarizes recent advances in LGMD pathogenesis and emerging therapeutic strategies, highlighting progress and remaining challenges in the field.
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Keywords
Pathological mechanism , Therapy strategy , Limb-girdle muscular dystrophy
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