Long-term treatment with the phosphorodiamidate morpholino oligomer eteplirsen in patients with Duchenne muscular dystrophy amenable to exon 51 skipping: analysis of dystrophin production
Titel:
Long-term treatment with the phosphorodiamidate morpholino oligomer eteplirsen in patients with Duchenne muscular dystrophy amenable to exon 51 skipping: analysis of dystrophin production
Auteur:
Charleston, J. Schnell, F.J. Dworzak, J. Donoghue, C. Lewis, S. Rodino-Klapac, L.R. Sahenk, Z. Voss, J. DeAlwis, U. Frank, D. Mendell, J.R.