Niemann-Pick disease type C1 is a sphingosine storage disease that causes deregulation of lysosomal calcium. by Lloyd-Evans, E, Morgan, A, He, X, Smith, D, Elliot-Smith, E, Sillence, D, Churchill, G, Schuchman, E, Galione, A, Platt, F

Central nervous system inflammation is a hallmark of pathogenesis in mouse models of GM1 and GM2 gangliosidosis. by Jeyakumar, M, Thomas, R, Elliot-Smith, E, Smith, D, van der Spoel, A, d'Azzo, A, Perry, V, Butters, T, Dwek, R, Platt, F

Beneficial effects of substrate reduction therapy in a mouse model of GM1 gangliosidosis. by Elliot-Smith, E, Speak, A, Lloyd-Evans, E, Smith, D, van der Spoel, A, Jeyakumar, M, Butters, T, Dwek, R, d'Azzo, A, Platt, F