Clinical characterisation of Becker muscular dystrophy patients predicts favourable outcome in exon-skipping therapy
J C van den Bergen, S M Schade van Westrum, L Dekker, A J van der Kooi, M de Visser, B H A Wokke, C S Straathof, M A Hulsker, A Aartsma-Rus, J J Verschuuren, H B Ginjaar
Journal of Neurology, Neurosurgery & Psychiatry Jan 2014, 85 (1) 92-98; DOI: 10.1136/jnnp-2012-304729