Phenotype of adult Refsum disease due to a defect in peroxin 7.

Horn, M A; van den Brink, D M; Wanders, R J A; Duran, M; Poll-The, B T; Tallaksen, C M E; Stokke, O H; Moser, H et al. · Neurology · 2007

case_report · Level V

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Abstract

The biochemical hallmark of adult Refsum disease (ARD) is an isolated deficiency in the breakdown of phytanic acid. This usually results from a PHYH gene defect, although some cases have been found to carry a PEX7 defect. We describe the phenotype of such a patient, indistinguishable from that of classic ARD. Hence, we propose the subdivision of ARD into type 1 and type 2, depending on which gene is defective.

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