Systemic administration of PRO051 in Duchenne's muscular dystrophy
Goemans, Nathalie M × Tulinius, Mar van den Akker, Johanna T Burm, Brigitte E Ekhart, Peter F Heuvelmans, Niki Holling, Tjadine Janson, Anneke A Platenburg, Gerard J Sipkens, Jessica A Sitsen, J M Ad Aartsma-Rus, Annemieke van Ommen, Gert-Jan B Buyse, Gunnar Darin, Niklas Verschuuren, Jan J Campion, Giles V de Kimpe, Sjef J van Deutekom, Judith C #
New England Journal of Medicine vol:364 issue:16 pages:1513-22
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 during pre-messenger RNA splicing of the dystrophin gene and to facilitate new dystrophin expression in muscle-fiber membranes. The present phase 1-2a study aimed to assess the safety, pharmacokinetics, and molecular and clinical effects of systemically administered PRO051.