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Article

Stereospecific rapid activation of Transcription Factor EB (TFEB) by Levacetylleucine (NALL)

2025-06-08

Abstract excerpt

N-acetyl L-leucine (NALL, USAN or levacetylleucine, INN or trade name Aqneursa™) is an FDA-approved agent for the treatment of Niemann-Pick disease type C (NPC). The N-acetyl group renders the compound a prodrug of L-leucine, making it a substrate for membrane-spanning monocarboxylate transporters (MCTs), which are ubiquitously expressed delivering NALL to all tissues with high capacity, including to the central n...

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Literature Corpus work
f7c0bb4d-f6f4-5bd4-badb-7d2b93d3cda0
DOI
10.1101/2025.06.06.657607
Open publication

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Stereospecific rapid activation of Transcription Factor EB (TFEB) by Levacetylleucine (NALL)DOI 10.1101/2025.06.06.657607
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