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... by Fields, T, Patterson, M, Bremova-Ertl, T, Belcher, G, Billington, I, Churchill, GC, 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, FM, Tsang, L, Verburg, C, Factor, M, Strupp, M