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

@article{Piscosquito2015ResponsivenessOC,
  title={Responsiveness of clinical outcome measures in Charcot-Marie-Tooth disease.},
  author={Giuseppe Piscosquito and Mary M Reilly and Angelo E Schenone and Gian Maria Fabrizi and Tiziana L Cavallaro and Lucio Santoro and Fiore Manganelli and Gianluca Emilio Ennio Vita and Aldo Quattrone and Luca Padua and Franco Gemignani and Francesco Visioli and Ma. Laura and Daniela Paola Calabrese and Richard Allen Hughes and D. Fernando Radice and Alessandra Solari and Davide Pareyson},
  journal={European journal of neurology},
  year={2015},
  volume={22 12},
  pages={
          1556-63
        }
}
BACKGROUND AND PURPOSE 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. METHODS The relative responsiveness of clinical scales of the Italian-UK ascorbic… CONTINUE READING
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