Acetyl-Leucine slows disease progression in lysosomal storage disorders - Kaya, E; Smith, DE; Smith, C; Morris, L; Bremova-Ertl, T; Cortina-Borja, M; Fineran, P; Morten, KJ; Poulton, J; Spencer, J; Strupp, ML; Platt, F (Wiley, 2020)