In June 2020, the article 'Brain Penetrable Inhibitors of Ceramide Galactosyltransferase for the Treatment of Lysosomal Storage Disorders' (https://pubs.acs.org/doi/10.1021/acsmedchemlett.0c00120) describing the discovery of "Compound 19" as a selective brain-penetrant inhibitor of the ceramide galactosyltransferase UGT8 was published. This drug candidate shows excellent oral PK/PD in the brain model and is intended for use in substrate reduction therapy for rare lysosomal storage disorders (e.g. Metachromatic Leukodystrophy, MLD). "Compound 19" contains thieno[3,2-b]pyridine moiety in its backbone.
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