Drug screening identifies tazarotene and bexarotene as therapeutic agents in multiple sulfatase deficiency
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DOI: 10.15252/emmm.202114837
Abstract: Multiple sulfatase deficiency (MSD, MIM #272200) results from pathogenic variants in the SUMF1 gene that impair proper function of the formylglycine‐generating enzyme (FGE). FGE is essential for the posttranslational activation of cellular sulfatases. MSD patients… read more here.
Keywords: tazarotene bexarotene; sulfatase; sulfatase deficiency; multiple sulfatase ... See more keywords