ACETYL-LEUCINE SLOWS DISEASE PROGRESSION IN LYSOSOMAL STORAGE DISORDERS

Acetyl-leucine improved symptoms of ataxia in particular in mice models and patients with the lysosomal storage disorders (LSD), Niemann-Pick disease type C  and GM2 Gangliosidosis. When N-acetyl-DL-leucine and N-acetyl-L-leucine were administered pre-symptomatically to Npc1-/- mice, both treatments delayed disease progression and extended life span, whereas ADL did not. These data are consistent with ALL being the neuroprotective enantiomer. Taken together, we have identified an unanticipated neuroprotective effect of acetyl-L-leucine and underlying mechanisms of action in LSDs, supporting its therapeutic potential for lysosomal disorders.

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