Litcius/Paper detail

<i>N</i> -Acetyl- <scp>l</scp> -Leucine and Neurodegenerative Disease

Cynthia J. Tifft

2024New England Journal of Medicine14 citationsDOI

Abstract

This editorial describes the science behind a study of N-acetyl-l-leucine to treat a specific lysosomal storage disorder, Niemann–Pick disease type C.

Topics & Concepts

LeucineDiseaseNeuroscienceChemistryBiochemistryBiologyMedicineAmino acidInternal medicineLysosomal Storage Disorders ResearchParkinson's Disease Mechanisms and TreatmentsGlycogen Storage Diseases and Myoclonus