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Hepatocyte transplantation (HTx) corrects selected neurometabolic abnormalities in murine intermediate maple syrup urine disease (iMSUD).
Biochim Biophys Acta. 2009 Oct;1792(10):1004-10. doi: 10.1016/j.bbadis.2009.08.006. Epub 2009 Aug 19.
Biochim Biophys Acta. 2009.
PMID: 19699299
Free PMC article.
Long chain fatty acid (Lcfa) abnormalities in hyper Igd syndrome (Hids) and Familial Mediterranean Fever (Fmf): new insight into heritable periodic fevers.
Simon A, Drenth JP, Matern D, Goetzman ES, Hager EJ, Gibson KM.
Simon A, et al.
Mol Genet Metab. 2013 Mar;108(3):166-71. doi: 10.1016/j.ymgme.2013.01.004. Epub 2013 Jan 15.
Mol Genet Metab. 2013.
PMID: 23375471
Free PMC article.
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