We took advantage of an ongoing observational study in which 13 NPC1 patients have been treated with TanganilTM (ADLL) continuously for ∼1 year and found that all patients showed stabilization or improvement in clinical scores, which were across all neurological domains, not just those relating to ataxia, supporting a more global neuroprotective effect in patients, analogous to those observed in the Npc1−/− mouse. Here, NPC1 is linked to cerebellar ataxia.