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Title: Ataluren treatment of patients with nonsense mutation dystrophinopathy
Authors: Bushby, Katharine ×
Finkel, Richard
Wong, Brenda
Barohn, Richard
Campbell, Craig
Comi, Giacomo P
Connolly, Anne M
Day, John W
Flanigan, Kevin M
Goemans, Nathalie
Jones, Kristi J
Mercuri, Eugenio
Quinlivan, Ros
Renfroe, James B
Russman, Barry
Ryan, Monique M
Tulinius, Mar
Voit, Thomas
Moore, Steven A
Lee Sweeney, H
Abresch, Richard T
Coleman, Kim L
Eagle, Michelle
Florence, Julaine
Gappmaier, Eduard
Glanzman, Allan M
Henricson, Erik
Barth, Jay
Elfring, Gary L
Reha, Allen
Spiegel, Robert J
O'donnell, Michael W
Peltz, Stuart W
Mcdonald, Craig M
Buyse, Gunnar
PTC124-GD-007-DMD STUDY GROUP #
Issue Date: Oct-2014
Publisher: John Wiley & Sons
Series Title: Muscle & Nerve vol:50 issue:4 pages:477-87
Article number: 10.1002/mus.24332
Abstract: Dystrophinopathy is a rare, severe muscle disorder, and nonsense mutations are found in 13% of cases. Ataluren was developed to enable ribosomal readthrough of premature stop codons in nonsense mutation (nm) genetic disorders.
URI: 
ISSN: 0148-639X
Publication status: published
KU Leuven publication type: IT
Appears in Collections:Organ Systems (+)
× corresponding author
# (joint) last author

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