Recent insights into limb-girdle muscular dystrophy: Impacts, therapy, and challenges

dc.contributor.authorJiang-Ling Xiao
dc.contributor.authorZhe Zhao
dc.contributor.authorHeng-Yuan Liu
dc.contributor.authorChang-Fa Tang
dc.contributor.authorChen-Chen Sun
dc.contributor.departmentBiología Celular e Histología
dc.date.accessioned2025-11-10T12:23:47Z
dc.date.available2025-11-10T12:23:47Z
dc.date.issued2025
dc.description.abstractLimb-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.
dc.formatapplication/pdf
dc.format.extent13
dc.identifier.doihttps://doi.org/10.14670/HH-18-929
dc.identifier.eissn1699-5848
dc.identifier.issn0213-3911
dc.identifier.urihttp://hdl.handle.net/10201/171911
dc.languageeng
dc.relationSin financiacion externa a la Universidad
dc.rightsAttribution-NonCommercial-NoDerivatives 4.0 International*
dc.rights.accessRightsinfo:eu-repo/semantics/openAccess
dc.rights.urihttp://creativecommons.org/licenses/by-nc-nd/4.0/*
dc.subjectPathological mechanism
dc.subjectTherapy strategy
dc.subjectLimb-girdle muscular dystrophy
dc.subject.odsNo relacionado con ningún objetivo de desarrollo sostenible
dc.titleRecent insights into limb-girdle muscular dystrophy: Impacts, therapy, and challenges
dc.typeinfo:eu-repo/semantics/article
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