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Mutations found in the MRP6 gene are associated with genetic abnormalities of the autosomal inherited connective tissue disorder called pseudoxanthoma elasticum (PXE), which is characterized by the presence of dystrophic elastic bers in the skin, retina, and large blood vessels, causing the appearance of bags in the skin, loss of vision and calcication of blood vessels [100]

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Thus, sodium dimethylbutyrate may be a promising alternative for those who cannot tolerate the side effects of hydroxycarbamide, which may include nausea, vomiting, diarrhea, constipation, and dizziness (Liebelt 2007)

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PubMed Central, Weinstock, Leonard B., et al

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