Ataluren treatment of patients with nonsense mutation dystrophinopathy
Muscle & Nerve2014Vol. 50(4), pp. 477–487
Citations Over TimeTop 1% of 2014 papers
K. Bushby, Richard S. Finkel, Brenda Wong, Richard J. Barohn, Craig Campbell, Giacomo P. Comi, Anne M. Connolly, John Day, Kevin M. Flanigan, Nathalie Goemans, Kristi Jones, Eugenio Mercuri, Rosaline C. M. Quinlivan, J. Ben Renfroe, Barry S. Russman, Monique M. Ryan, M. Tulinius, Thomas Voït, Steven A. Moore, H. Lee Sweeney, Richard T. Abresch, Kim L. Coleman, Michelle Eagle, Julaine Florence, Eduard Gappmaier, Allan M. Glanzman, Erik Henricson, Jay Barth, Gary Elfring, A. Reha, Robert J. Spiegel, Michael W. O'donnell, Stuart W. Peltz, Craig M. McDonald, FOR THE PTC124‐GD‐007‐DMD STUDY GROUP
Abstract
As the first investigational new drug targeting the underlying cause of nm-dystrophinopathy, ataluren offers promise as a treatment for this orphan genetic disorder with high unmet medical need.
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