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https://pubmed.ncbi.nlm.nih.gov/38113551
This study found that single and multiple doses of the experimental drug nizubaglustat, a dual inhibitor of ceramide glucosyltransferase and non-lysosomal neutral glucosylceramidase, were safe and well-tolerated in healthy adults, and demonstrated target engagement and pharmacodynamic effects indicating potential for the treatment of neurological manifestations in primary and secondary gangliosidoses.