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