Following the precedent created by small molecular weight compounds targeting the cystic fibrosis transmembrane conductance regulator (CFTR) channel, scientists at the University of Munich have targeted TRPML1, the endolysosomal cation channel mutated in Mucolipidosis type IV.202 The authors identified a small molecule that restores the phenotype of a specific subset of TRPML1 mutations. The gene discussed is MCOLN1; the disease is mucolipidosis type IV.