D01 Golodirsen induces exon skipping leading to sarcolemmal dystrophin expression in patients with genetic mutations amenable to exon 53 skipping
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E. Mercuri | F. Muntoni | L. Servais | L. Popplewell | G. Dickson | V. Straub | M. Guglieri | J. Domingos | M. Monforte | J. Morgan | A. Seferian | D. Frank | F. Schnell