Given the known effects of long-term or permanent loss/inhibition of TRPML1 in humans, resulting in the lysosomal storage disorder (LSD) mucolipidosis type IV with a severe neurodegenerative phenotype and severely reduced life span, TRPML1 inhibition seems not to be a particularly safe option, especially in younger patients. This evidence concerns the gene MCOLN1 and lysosomal storage disease.