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Cardiac pathology exceeds skeletal muscle pathology in two cases of limb-girdle muscular dystrophy type 2I
Journal article   Open access   Peer reviewed

Cardiac pathology exceeds skeletal muscle pathology in two cases of limb-girdle muscular dystrophy type 2I

Marta Margeta, Anne M Connolly, Thomas L Winder, Alan Pestronk and Steven A Moore
Muscle & nerve, Vol.40(5), pp.883-889
11/2009
DOI: 10.1002/mus.21432
PMCID: PMC2862182
PMID: 19705481
url
https://www.ncbi.nlm.nih.gov/pmc/articles/2862182View
Open Access

Abstract

Limb-girdle muscular dystrophy type 2I (LGMD-2I) is caused by mutations in the fukutin-related protein gene (FKRP) that lead to abnormal glycosylation of alpha-dystroglycan in skeletal muscle. Heart involvement in LGMD-2I is common, but little is known about a underlying cardiac pathology. Herein we describe two patients with LGMD-2I (homozygous FKRP mutation c.826C>A, p.Leu276Ile) who developed severe congestive heart failure that required cardiac transplantation. The dystrophic pathology and impairment of alpha-dystroglycan glycosylation were severe in the heart but mild in skeletal muscle, underscoring the lack of correlation between cardiac and skeletal muscle involvement in some LGMD-2I patients.
Muscular Dystrophies, Limb-Girdle - classification Humans Adolescent Female Male Heart Failure - pathology Muscle, Skeletal - pathology Muscular Dystrophies, Limb-Girdle - pathology Child

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