THE EFECTS OF N‑ACETYL‑L‑LEUCINE ON THE IMPROVEMENT OF SYMPTOMS IN A PATIENT WITH MULTIPLE SULFATASE DEFCIENCY

In this placebo-controlled, crossover case study, a 12 year old patient with Multiple Sulfatase Deficiency was treated with N-acetyl-L-leucine (NALL) and placebo in two 1-month periods. Treatment with NALL was well tolerated and significantly improved symptoms (including the SARA and SCAFI scores), quality of life measure, and serum IL-6 levels, a biomarker for neuroinflammation.

Regarding quality of life assessments, the parent and child reported improved quality of life index, physical health, and emotional function after taking NALL. Moreover, total energy intake was increased with NALL treat- ment through the study period. The Cerebellum.

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