Patients with Niemann-Pick disease type C who received N-acetyl-L-leucine (NALL), an agent that potentially ameliorates lysosomal and metabolic dysfunction, showed reduced neurologic signs and ...
Niemann–Pick disease type C, a lysosomal storage disease, causes progressive neurologic impairment. N-acetyl-l-leucine normalizes cellular energy production and improves lysosomal function. New ...
In Niemann–Pick disease type C, a rare lysosomal storage disorder, treatment with N-acetyl-l-leucine, which may ameliorate lysosomal and metabolic dysfunction, resulted in better neurologic status ...
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