Article
Systemic administration of PRO051 in Duchenne's muscular dystrophy.
The New England journal of medicine - 21 Apr 2011
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
Abstract excerpt
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 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,...
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