N-Acetyl-l-Leucine for Niemann-Pick Type C: Cautious Optimism for an Expanded Treatment Toolkit

Neurology. 2025 Jul;105(1):e213857. doi: 10.1212/WNL.0000000000213857. Epub 2025 Jun 13.

NO ABSTRACT

PMID:40513056 | DOI:10.1212/WNL.0000000000213857