PPT1 and neuronal ceroid lipofuscinosis: Recently, a clinical trial (ClinicalTrials.gov identifier NCT00028262) for the treatment of patients with infantile NCL associated with mutations in the CLN1 gene encoding for palmitoyl-protein thioesterase-1 has been carried out by orally administering the nucleophilic small molecules cysteamine bitartrate and N-acetylcysteine, aiming at the reduction of ceroid lipofuscin and granular osmiophilic deposits (GROD) (Levin et al., 2014).