Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6–48 months with Duchenne muscular dystrophy amenable to exon 51 skipping
- Resource Type
- Article
- Authors
- Deconinck, Nicolas; Van Coster, Rudy; Vanlander, Arnaud; Seferian, Andreea; De Lucia, Silvana; Gidaro, Teresa; Brande, Laura Vanden; Servais, Laurent; Kirschner, Janbernd; Borell, Sabine; Mercuri, Eugenio; Brogna, Claudia; Pane, Marika; Fanelli, Lavinia; Norcia, Giulia; Muntoni, Francesco; Brusa, Chiara; Chesshyre, Mary; Maresh, Kate; Pitchforth, Jaqueline; Schottlaender, Lucia; Scoto, Mariacristina; Silwal, Arpana; Trucco, Fedrica; Mercuri, E.; Seferian, A.M.; Servais, L.; Deconinck, N.; Stevenson, H.; Ni, X.; Zhang, W.; East, L.; Yonren, S.; Muntoni, F.
- Source
- In Neuromuscular Disorders June 2023 33(6):476-483
- Subject
- Language
- ISSN
- 0960-8966