Systemic administration of PRO051 in Duchenne’s muscular dystrophy

Goemans NM, Tulinius M, van den Akker JT, et al. Systemic administration of PRO051 in Duchenne’s muscular dystrophy. The New England journal of medicine 2011;364:1513-22   Abstract BACKGROUND: Local intramuscular administration of the antisense oligonucleotide PRO051 in patients with Duchenne’s muscular dystrophy with relevant mutations was previously reported to induce the skipping of exon 51 […]