Golodirsen induces exon skipping leading to sarcolemmal dystrophin expression in patients with genetic mutations amenable to exon 53 skipping
Muntoni, F., Frank, D.E., Morgan, J., Domingos, J., Schnell, F.J., Dickson, G., Popplewell, L., Guglieri, M., Seferian, A., Monforte, M., Mercuri, E., Servais, L., Straub, V.Volume:
28
Language:
english
Journal:
Neuromuscular Disorders
DOI:
10.1016/S0960-8966(18)30304-3
Date:
April, 2018
File:
PDF, 25 KB
english, 2018