Ascorbic acid in Charcot-Marie-Tooth disease type 1A (CMT-TRIAAL and CMT-TRAUK): a double-blind randomised trial

Pareyson, D; Reilly, Mm; Schenone, A; Fabrizi, Gm; Cavallaro, T; Santoro, Lucio; Vita, G; Quattrone, A; Padua, L; Gemignani, F; Visioli, F; Laurà, M; Radice, D; Calabrese, D; Hughes, Ra; Solari, A; Pareyson, D; Marchesi, C; Salsano, E; Nanetti, L; Marelli, C; Scaioli, V; Ciano, C; Rimoldi, M; Lauria, G; Rizzetto, E; Camozzi, F; Schenone, A; Narciso, E; Grandis, M; Monti Bragadin, M; Nobbio, L; Fabrizi, Gm; Cavallaro, T; Casano, A; Bertolasi, L; Cabrini, I; Corrà, K; Rizzuto, N; Santoro, L; Manganelli, Fiore; Pisciotta, Chiara; Nolano, M; Vita, G; Mazzeo, A; Aguennouz, M; Di Leo, R; Majorana, G; Lanzano, N; Valenti, F; Quattrone, A; Valentino, P; Nisticò, R; Pirritano, D; Lucisano, A; Canino, M; Padua, L; Pazzaglia, C; Granata, G; Foschini, M; Gemignani, F; Brindani, F; Vitetta, F; Allegri, I; Visioli, F; Bogani, P; Visioli, F; Reilly, Mm; Laurà, M; Blake, J; Koltzenburg, M; Hutton, E; Lunn, M; Hughes, Ra; Pareyson, D; Solari, A; Schenone, A; Visioli, F; Reilly, Mm; Mancardi, Gl; Solari, A; Cavaletti, G; Galimberti, S; Solari, A; Radice, D; Calabrese, D; Ferrari, G; Visioli, F; Rimoldi, M; Scaioli, V; Santoro, L; Padua, L; Schenone, A; Santoro, L; Lauria, G; Sereda, M.; Nolano, Maria

doi:10.1016/S1474-4422(11)70025-4

Ascorbic acid reduced the severity of neuropathy in transgenic mice overexpressing peripheral myelin protein 22 (PMP22), a model of Charcot-Marie-Tooth disease type 1A (CMT1A) associated with the PMP22 duplication. However, in three 1-year trials, ascorbic acid had no benefit in human beings. We did a multicentre 2-year trial to test the efficacy and tolerability of ascorbic acid in patients with CMT1A.

Ascorbic acid in Charcot-Marie-Tooth disease type 1A (CMT-TRIAAL and CMT-TRAUK): a double-blind randomised trial / Pareyson, D; Reilly, Mm; Schenone, A; Fabrizi, Gm; Cavallaro, T; Santoro, Lucio; Vita, G; Quattrone, A; Padua, L; Gemignani, F; Visioli, F; Laurà, M; Radice, D; Calabrese, D; Hughes, Ra; Solari, A; Pareyson, D; Marchesi, C; Salsano, E; Nanetti, L; Marelli, C; Scaioli, V; Ciano, C; Rimoldi, M; Lauria, G; Rizzetto, E; Camozzi, F; Schenone, A; Narciso, E; Grandis, M; Monti Bragadin, M; Nobbio, L; Fabrizi, Gm; Cavallaro, T; Casano, A; Bertolasi, L; Cabrini, I; Corrà, K; Rizzuto, N; Santoro, L; Manganelli, Fiore; Pisciotta, Chiara; Nolano, M; Vita, G; Mazzeo, A; Aguennouz, M; Di Leo, R; Majorana, G; Lanzano, N; Valenti, F; Quattrone, A; Valentino, P; Nisticò, R; Pirritano, D; Lucisano, A; Canino, M; Padua, L; Pazzaglia, C; Granata, G; Foschini, M; Gemignani, F; Brindani, F; Vitetta, F; Allegri, I; Visioli, F; Bogani, P; Visioli, F; Reilly, Mm; Laurà, M; Blake, J; Koltzenburg, M; Hutton, E; Lunn, M; Hughes, Ra; Pareyson, D; Solari, A; Schenone, A; Visioli, F; Reilly, Mm; Mancardi, Gl; Solari, A; Cavaletti, G; Galimberti, S; Solari, A; Radice, D; Calabrese, D; Ferrari, G; Visioli, F; Rimoldi, M; Scaioli, V; Santoro, L; Padua, L; Schenone, A; Santoro, L; Lauria, G; Sereda, M.; Nolano, Maria. - In: LANCET NEUROLOGY. - ISSN 1474-4422. - 10:4(2011), pp. 320-8-328. [10.1016/S1474-4422(11)70025-4]