l-NBDNJ allosteric chaperone for multiple defective enzymes involved in lysosomal storage disorders (LSDs).

miglustat - Generic mfg.

Plicera (afegostat tartrate) - BioMarin

http://www.ncbi.nlm.nih.gov/pubmed/42107261

Alejandro Montesa; Anna Esposito; Annalisa Guaragna; Atsushi Kato; Eliana De Gregorio; Kosuke Yoshimura; Maria Stabile; Pedro Merino; Uta Kanekiyo

May 11, 2026