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1.	Bushby, Katharine, et al. (author) Ataluren treatment of patients with nonsense mutation dystrophinopathy. 2014 In: Muscle & nerve. - : Wiley. - 1097-4598 .- 0148-639X. ; 50:4, s. 477-87 Journal article (peer-reviewed)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.
2.	Goemans, Nathalie M, et al. (author) Systemic administration of PRO051 in Duchenne's muscular dystrophy. 2011 In: The New England journal of medicine. - 1533-4406. ; 364:16, s. 1513-22 Journal article (other academic/artistic)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.

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Author/Editor: Tulinius, Mar, 1953 (2); Darin, Niklas, 1964 (1); Aartsma-Rus, Annemie ... (1); Moore, Steven A (1); Campbell, Craig (1); Flanigan, Kevin M. (1); show more...; van Ommen, Gert-Jan ... (1); Bushby, Katharine (1); Finkel, Richard (1); Wong, Brenda (1); Barohn, Richard (1); Comi, Giacomo P (1); Connolly, Anne M (1); Day, John W (1); Goemans, Nathalie (1); Jones, Kristi J (1); Mercuri, Eugenio (1); Quinlivan, Ros (1); Renfroe, James B (1); Russman, Barry (1); Ryan, Monique M (1); Voit, Thomas (1); Lee Sweeney, H (1); Abresch, Richard T (1); Coleman, Kim L (1); Eagle, Michelle (1); Florence, Julaine (1); Gappmaier, Eduard (1); Glanzman, Allan M (1); Henricson, Erik (1); Barth, Jay (1); Elfring, Gary L (1); Reha, Allen (1); Spiegel, Robert J (1); O'donnell, Michael W (1); Peltz, Stuart W (1); McDonald, Craig M (1); Buyse, Gunnar (1); Goemans, Nathalie M (1); van den Akker, Johan ... (1); Burm, Brigitte E (1); Ekhart, Peter F (1); Heuvelmans, Niki (1); Holling, Tjadine (1); Janson, Anneke A (1); Platenburg, Gerard J (1); Sipkens, Jessica A (1); Sitsen, J M Ad (1); Verschuuren, Jan J (1); Campion, Giles V (1); show less...

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