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Title: Systemic administration of PRO051 in Duchenne's muscular dystrophy
Authors: 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 #
Issue Date: Apr-2011
Series Title: New England Journal of Medicine vol:364 issue:16 pages:1513-22
Abstract: 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.
URI: 
ISSN: 0028-4793
Publication status: published
KU Leuven publication type: IT
Appears in Collections:Brain & Metabolism Section (-)
× corresponding author
# (joint) last author

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