Treatment with miglustat reverses the lipid-trafficking defect in Niemann-Pick disease type C. by Lachmann, R, te Vruchte, D, Lloyd-Evans, E, Reinkensmeier, G, Sillence, D, Fernandez-Guillen, L, Dwek, R, Butters, T, Cox, T, Platt, F

Substrate reduction therapy in the infantile form of Tay-Sachs disease. by Bembi, B, Marchetti, F, Guerci, V, Ciana, G, Addobbati, R, Grasso, D, Barone, R, Cariati, R, Fernandez-Guillen, L, Butters, T, Pittis, MG