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The nucleotide prodrug CERC-913 improves mtDNA content in primary hepatocytes from DGUOK-deficient rats.
J Inherit Metab Dis. 2021 Mar;44(2):492-501. doi: 10.1002/jimd.12354. Epub 2021 Jan 15.
J Inherit Metab Dis. 2021.
PMID: 33368311
Targeting GNE Myopathy: A Dual Prodrug Approach for the Delivery of N-Acetylmannosamine 6-Phosphate.
Morozzi C, Sedláková J, Serpi M, Avigliano M, Carbajo R, Sandoval L, Valles-Ayoub Y, Crutcher P, Thomas S, Pertusati F.
Morozzi C, et al. Among authors: crutcher p.
J Med Chem. 2019 Sep 12;62(17):8178-8193. doi: 10.1021/acs.jmedchem.9b00833. Epub 2019 Aug 20.
J Med Chem. 2019.
PMID: 31386361
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