Electrocardiogram in Friedreich's ataxia: A shortâterm surrogate endpoint for treatment efficacy
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DOI: 10.1111/anec.12813
Abstract: Friedreich's ataxia is a rare degenerative neuromuscular disorder, caused by a homozygous GAA triplet repeat expansion in the frataxin (FXN) gene, with a broad clinical phenotype characterized by progressive gait and limb ataxia, dysarthria, and… read more here.
Keywords: term; friedreich ataxia; electrocardiogram friedreich; treatment ... See more keywords