N-Acetyl-L-Leucine improves neurological outcomes in Niemann-Pick Disease type C
1. In this randomized controlled trial, N-acetyl-L-leucine (NALL) improved neurologic status in Niemann-Pick Disease type C more effectively than a placebo. 2. Patients showed a significant reduction in neurological symptoms after taking NALL. Evidence Rating Level: 1 (Excellent) Study Rundown: Niemann-Pick Disease Type C is an inherited lysosomal storage disorder following the autosomal recessive pattern