Disease-Modifying, Neuroprotective Effect of N-Acetyl-
l
-Leucine in Adult and Pediatric Patients With Niemann-Pick Disease Type C
Neurology | June 13, 2025 | Volume: 105 | Issue: 1 | Pages: No page available
10.1212/wnl.0000000000213589
| PMID 40513057