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Fields, T; Bremova, T M; Billington, I; Churchill, G C; Evans, W; Fields, C; Galione, A; Kay, R; Mathieson, T; Martakis, K; Patterson, M; Platt, F; Factor, M; Strupp, M (2023). N-acetyl-L-leucine for Niemann-Pick type C: a multinational double-blind randomized placebo-controlled crossover study. Trials, 24(1), p. 361. BioMed Central 10.1186/s13063-023-07399-6
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; ... (2021). 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 telangiectasia. Trials, 22(1), p. 84. BioMed Central 10.1186/s13063-020-05009-3
