GNE mutations causing distal myopathy with rimmed vacuoles with inflammation.

Yabe, I; Higashi, T; Kikuchi, S; Sasaki, H; Fukazawa, T; Yoshida, K; Tashiro, K · Neurology · 2003

case_report · Level V

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Abstract

The authors describe a family in which two individuals have clinical distal myopathy with rimmed vacuoles (DMRV). While the clinical and most of the pathologic features in these patients were compatible with a diagnosis of DMRV, the presence of inflammatory changes in the connective tissue between muscle fibers was not. Gene analysis revealed a compound heterozygous mutation in these individuals, characterized by V572L and I472T.

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