“Safety, Tolerability, and Pharmacodynamics of Experimental Drug Nizubaglustat in Healthy Adults”

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.