Responsiveness of clinical outcome measures in Charcot-Marie-Tooth disease

Creators: Piscosquito G; Reilly MM; Schenone A; Fabrizi GM; Cavallaro T; Santoro L; Manganelli F; Vita G; Quattrone A; Padua L; Gemignani F; Visioli F; Laurà M; Calabrese D; Hughes RA; Radice D; Solari A; Pareyson D.

Others:: Piscosquito, G; Reilly, Mm; Schenone, A; Fabrizi, Gm; Cavallaro, T; Santoro, L; Manganelli, F; Vita, G; Quattrone, A; Padua, L; Gemignani, F; Visioli, F; Laurà, M; Calabrese, D; Hughes, Ra; Radice, D; Solari, A; Pareyson, D.

Description

Charcot-Marie-Tooth disease (CMT) is a very slowly progressive neuropathy which makes it difficult to detect disease progression over time and to assess intervention efficacy. Experience from completed clinical trials with ascorbic acid and natural history studies confirm difficulties in detecting such changes. Consequently, sensitive-to-change outcome measures (OMs) are urgently needed.

Responsiveness of clinical outcome measures in Charcot-Marie-Tooth disease

Description

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