Article

N-acetyl-L-leucine (Levacetylleucine) normalizes Transcription Factor EB (TFEB) activity by stereospecific bidirectional modulation

2025-12-02

Abstract excerpt

Levacetylleucine (Aqneursa TM ), a chemically modified amino acid, is the only US Food and Drug Administration-approved monotherapy for the treatment of Niemann-Pick disease type C (NPC) (Beninger, 2024; Mullard, 2024; van Gool et al., 2025). This acetylated derivative of L-leucine functions as a pro-drug, with the acetyl group rendering it a substrate for the monocarboxylate transporter (MCT) family of transport...

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Literature Corpus work: 3bc07a26-ec91-5360-9575-7db5af50349b
DOI: 10.64898/2025.11.30.691375

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