N-acetyl-L-leucine for Niemann-Pick type C: a multinational double-blind randomized placebo-controlled crossover study

por Fields, T, M. Bremova, T, Billington, I, Churchill, GC, Evans, W, Fields, C, Galione, A, Kay, R, Mathieson, T, Martakis, K, Patterson, M, Platt, F, Factor, M, Strupp, M
Publicado 2023