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Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy by Frank, DE, Schnell, FJ, Akana, C, El-Husayni, SH, Desjardins, CA, Morgan, J, Charleston, JS, Sardone, V, Domingos, J, Dickson, G, Straub, V, Guglieri, M, Mercuri, E, Servais, L, Muntoni, F
Published 2020Journal article -
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Delivery is key: lessons learnt from developing splice-switching antisense therapies by Godfrey, C, Desviat, L, Smedsrød, B, Piétri-Rouxel, F, Denti, M, Disterer, P, Lorain, S, Nogales-Gadea, G, Sardone, V, Anwar, R, El Andaloussi, S, Lehto, T, Khoo, B, Brolin, C, , v, Goyenvalle, A, Aartsma-Rus, A, Arechavala-Gomeza, V
Published 2017Journal article