Myopathy With Exercise-Induced Intolerance due to Novel Biallelic Variants in OBSCN-A Clinical, Morphological and Molecular Analysis

由OBSCN-A基因新型双等位基因变异引起的运动诱发性肌病:临床、形态学和分子分析

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Abstract

The phenotype of OBSCN variants consists of exercise intolerance ranging from myalgia and cramps to rhabdomyolysis. Symptoms are mainly induced by high-intensity sports. Molecular analysis showing a deregulation of muscle processes associated with Ca(2+) regulation, extrasarcolemmal integrity and autophagy emphasised the critical role of obscurin in skeletal muscle function.

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