Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6–48 months with Duchenne muscular dystrophy amenable to exon 51 skipping

Eugenio Mercuri, A. Seferian, Laurent Servais, Nicolas Deconinck, H. Stevenson, X. Ni, W. Zhang, Lilly East, S. Yonren, Francesco Muntoni, Nicolas Deconinck, Rudy Van Coster, Arnaud Vanlander, A. Seferian, Silvana De Lucia, Teresa Gidaro, Laura Vanden Brande, Laurent Servais, Janbernd Kirschner, S. Borell, Eugenio Mercuri, Claudia Brogna, Marika Pane, Lavinia Fanelli, Giulia Norcia, Francesco Muntoni, Chiara Brusa, Mary Chesshyre, Kate Maresh, J. Pitchforth, Lucía Schottlaender, Mariacristina Scoto, Arpana Silwal, Fedrica Trucco