Title: | Clinical characterisation of Becker muscular dystrophy patients predicts favourable outcome in exon-skipping therapy |
Journal : | Journal of neurology, neurosurgery and psychiatry, 85, 1 |
Authors: | van den Bergen JC ; Schade van Westrum SM ; Dekker L ; van der Kooi AJ ; de Visser M ; Wokke BHA ; Straathof CS ; Hulsker MA ; Aartsma Rus A ; Verschuuren JJ ; Ginjaar HB |
Material Type: | Article |
Publication Date: | 01/2014 |
Size: | p. 92-98 |
Languages: | English |
Keywords : | Becker muscular dystrophy ; cardiac features ; clinical presentation ; cohort studies ; database ; death ; dilated cardiomyopathy ; disease evolution ; Duchenne muscular dystrophy ; exon skipping ; genetic mutations ; Netherlands ; wheelchairs |
Pubmed / DOI : | DOI : 10.1136/jnnp-2012-304729 / Pubmed : 23667215 |