Of note, TRPML1 activation rescues dysfunctional phenotype in lysosomal storage diseases, including Neuronal Ceroid Lipofuscinoses (NCL, Batten disease), mucopolysaccharidoses, such as Hurler or Hunter syndrome, sphingolipidoses, such as Fabry, Gaucher or Niemann–Pick-type C1 (NPC1), and mucolipidoses, such as Mucolipidosis type IV (MLIV), characterized by a greater neurodegenerative component [46-51]. This evidence concerns the gene MCOLN1 and mucolipidosis type IV.