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

A master protocol to investigate a novel therapy acetyl-l-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosidoses, and ataxia telangiec...

por Fields, T., Patterson, M., Bremova-Ertl, T., Belcher, G., Billington, I., Churchill, G. C., Davis, W., Evans, W., Flint, S., Galione, A., Granzer, U., Greenfield, J., Karl, R., Kay, R., Lewi, D., Mathieson, T., Meyer, T., Pangonis, D., Platt, F. M., Tsang, L., Verburg, C., Factor, M., Strupp, M.
Publicado 2021