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ArticlesPeer ReviewedFull Text
Ataluren: First Global Approval
Author / Creator:Ryan, Nicola J.
Publisher:Cham: Springer International Publishing
Journal title:Drugs (New York, N.Y.), 2014-09, Vol.74 (14), p.1709-1714
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Ataluren
Author / Creator:Kaufman, Michele B
Publisher:Intellisphere, LLC
Journal title:Formulary (Cleveland, Ohio), 2011-02, Vol.46 (2), p.68
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Long term treatment with ataluren—the Swedish experience
Author / Creator:Michael, Eva ; Sofou, Kalliopi ; Wahlgren, Lisa ; Kroksmark, Anna-Karin ; Tulinius, Már
Publisher:London: BioMed Central Ltd
Journal title:BMC musculoskeletal disorders, 2021-09, Vol.22 (1), p.1-837, Article 837
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Effect of Ataluren on dystrophin mutations
Author / Creator:Berger, Joachim ; Li, Mei ; Berger, Silke ; Meilak, Michelle ; Rientjes, Jeanette ; Currie, Peter D.
Publisher:England: John Wiley & Sons, Inc
Journal title:Journal of cellular and molecular medicine, 2020-06, Vol.24 (12), p.6680-6689
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Ataluren—Promising Therapeutic Premature Termination Codon Readthrough Frontrunner
Author / Creator:Michorowska, Sylwia
Publisher:Basel: MDPI AG
Journal title:Pharmaceuticals (Basel, Switzerland), 2021-08, Vol.14 (8), p.785
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ArticlesPeer ReviewedFull Text
Ataluren stimulates ribosomal selection of near-cognate tRNAs to promote nonsense suppression
Author / Creator:Roy, Bijoyita ; Friesen, Westley J. ; Tomizawa, Yuki ; Leszyk, John D. ; Zhuo, Jin ; Johnson, Briana ; Dakka, Jumana ; Trotta, Christopher R. ; Xue, Xiaojiao ; Mutyam, Venkateshwar ; Keeling, Kim M. ; Mobley, James A. ; Rowe, Steven M. ; Bedwell, David M. ; Welch, Ellen M. ; Jacobson, Allan
Publisher:United States: National Academy of Sciences
Journal title:Proceedings of the National Academy of Sciences - PNAS, 2016-11, Vol.113 (44), p.12508-12513
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Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
Author / Creator:McDonald, Craig M ; Campbell, Craig ; Torricelli, Ricardo Erazo ; Finkel, Richard S ; Flanigan, Kevin M ; Goemans, Nathalie ; Heydemann, Peter ; Kaminska, Anna ; Kirschner, Janbernd ; Muntoni, Francesco ; Osorio, Andrés Nascimento ; Schara, Ulrike ; Sejersen, Thomas ; Shieh, Perry B ; Sweeney, H Lee ; Topaloglu, Haluk ; Tulinius, Már ; Vilchez, Juan J ; Voit, Thomas ; Wong, Brenda ; Elfring, Gary ; Kroger, Hans ; Luo, Xiaohui ; McIntosh, Joseph ; Ong, Tuyen ; Riebling, Peter ; Souza, Marcio ; Spiegel, Robert J ; Peltz, Stuart W ; Mercuri, Eugenio ; Alfano, Lindsay N ; Eagle, Michelle ; James, Meredith K ; Lowes, Linda ; Mayhew, Anna ; Mazzone, Elena S ; Nelson, Leslie ; Rose, Kristy J ; Abdel-Hamid, Hoda Z ; Apkon, Susan D ; Barohn, Richard J ; Bertini, Enrico ; Bloetzer, Clemens ; de Vaud, Lausanne Canton ; Butterfield, Russell J ; Chabrol, Brigitte ; Chae, Jong-Hee ; Jongno-gu, Daehak-ro ; Comi, Giacomi Pietro ; Darras, Basil T ; Dastgir, Jahannaz ; Desguerre, Isabelle ; Escobar, Raul G ; Finanger, Erika ; Guglieri, Michela ; Hughes, Imelda ; Iannaccone, Susan T ; Jones, Kristi J ; Karachunski, Peter ; Kudr, Martin ; Lotze, Timothy ; Mah, Jean K ; Mathews, Katherine ; Nevo, Yoram ; Parsons, Julie ; Péréon, Yann ; de Queiroz Campos Araujo, Alexandra Prufer ; Renfroe, J Ben ; de Resende, Maria Bernadete Dutra ; Ryan, Monique ; Selby, Kathryn ; Tennekoon, Gihan ; Vita, Giuseppe
Publisher:England: Elsevier Ltd
Journal title:The Lancet (British edition), 2017-09, Vol.390 (10101), p.1489-1498
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ArticlesPeer ReviewedFull Text
LC–MS/MS quantification of ataluren and ataluren acyl glucuronide in human plasma/urine: application in clinical studies
Author / Creator:Kaushik, Diksha ; Ma, Jiyuan ; Gu, Guodong ; Hwang, Seongwoo ; Moon, Young-Choon ; Kong, Ronald
Publisher:Newlands Press Ltd
Journal title:Bioanalysis, 2020-11, Vol.12 (21), p.1545-1555
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ArticlesPeer ReviewedFull Text
Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy
Author / Creator:Finkel, Richard S ; Flanigan, Kevin M ; Wong, Brenda ; Bönnemann, Carsten ; Sampson, Jacinda ; Sweeney, H Lee ; Reha, Allen ; Northcutt, Valerie J ; Elfring, Gary ; Barth, Jay ; Peltz, Stuart W
Publisher:United States: Public Library of Science
Journal title:PloS one, 2013-12, Vol.8 (12), p.e81302-e81302
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ArticlesPeer ReviewedFull Text
Proposing a mechanism of action for ataluren
Author / Creator:Siddiqui, Nadeem ; Sonenberg, Nahum
Publisher:United States: National Academy of Sciences
Journal title:Proceedings of the National Academy of Sciences - PNAS, 2016-11, Vol.113 (44), p.12353-12355
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ArticlesPeer ReviewedFull Text
Ataluren and aminoglycosides stimulate read-through of nonsense codons by orthogonal mechanisms
Author / Creator:Ng, Martin Y. ; Li, Hong ; Ghelfi, Mikel D. ; Goldman, Yale E. ; Cooperman, Barry S.
Publisher:United States: National Academy of Sciences
Journal title:Proceedings of the National Academy of Sciences - PNAS, 2021-01, Vol.118 (2), p.1-10
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Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015–2022): 2022 interim analysis
Author / Creator:Mercuri, Eugenio ; Osorio, Andrés Nascimento ; Muntoni, Francesco ; Buccella, Filippo ; Desguerre, Isabelle ; Kirschner, Janbernd ; Tulinius, Már ; de Resende, Maria Bernadete Dutra ; Morgenroth, Lauren P. ; Gordish-Dressman, Heather ; Johnson, Shelley ; Kristensen, Allan ; Werner, Christian ; Trifillis, Panayiota ; Henricson, Erik K. ; McDonald, Craig M.
Publisher:Berlin/Heidelberg: Springer Berlin Heidelberg
Journal title:Journal of neurology, 2023-08, Vol.270 (8), p.3896-3913
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ArticlesPeer ReviewedFull Text
Ataluren binds to multiple protein synthesis apparatus sites and competitively inhibits release factor-dependent termination
Author / Creator:Huang, Shijie ; Bhattacharya, Arpan ; Ghelfi, Mikel D. ; Li, Hong ; Fritsch, Clark ; Chenoweth, David M. ; Goldman, Yale E. ; Cooperman, Barry S.
Publisher:London: Nature Publishing Group UK
Journal title:Nature communications, 2022-05, Vol.13 (1), p.2413-14, Article 2413
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Correction of nonsense BMPR2 and SMAD9 mutations by ataluren in pulmonary arterial hypertension
Author / Creator:Drake, Kylie M ; Dunmore, Benjamin J ; McNelly, Lauren N ; Morrell, Nicholas W ; Aldred, Micheala A
Publisher:United States: American Thoracic Society
Journal title:American journal of respiratory cell and molecular biology, 2013-09, Vol.49 (3), p.403-409
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Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study
Author / Creator:Mercuri, Eugenio ; Muntoni, Francesco ; Osorio, Andrés Nascimento ; Tulinius, Már ; Buccella, Filippo ; Morgenroth, Lauren P ; Gordish-Dressman, Heather ; Jiang, Joel ; Trifillis, Panayiota ; Zhu, Jin ; Kristensen, Allan ; Santos, Claudio L ; Henricson, Erik K ; McDonald, Craig M ; Desguerre, Isabelle
Publisher:England: Future Medicine Ltd
Journal title:Journal of comparative effectiveness research, 2020-04, Vol.9 (5), p.341-360
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Ataluren suppresses a premature termination codon in an MPS I-H mouse
Author / Creator:Wang, Dan ; Xue, Xiaojiao ; Gunn, Gwen ; Du, Ming ; Siddiqui, Amna ; Weetall, Marla ; Keeling, Kim M.
Publisher:Berlin/Heidelberg: Springer Berlin Heidelberg
Journal title:Journal of molecular medicine (Berlin, Germany), 2022-08, Vol.100 (8), p.1223-1235
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Ataluren for the Treatment of Usher Syndrome 2A Caused by Nonsense Mutations
Author / Creator:Samanta, Ananya ; Stingl, Katarina ; Kohl, Susanne ; Ries, Jessica ; Linnert, Joshua ; Nagel-Wolfrum, Kerstin
Publisher:Switzerland: MDPI AG
Journal title:International journal of molecular sciences, 2019-12, Vol.20 (24), p.6274
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Ataluren, a New Therapeutic for Alpha-1 Antitrypsin-Deficient Individuals with Nonsense Mutations
Author / Creator:Reeves, Emer P ; O'Dwyer, Ciara A ; Dunlea, Danielle M ; Wormald, Mark R ; Hawkins, Padraig ; Alfares, Mohammad ; Kotton, Darrell N ; Rowe, Steven M ; Wilson, Andrew A ; McElvaney, Noel G
Publisher:United States: American Thoracic Society
Journal title:American journal of respiratory and critical care medicine, 2018-10, Vol.198 (8), p.1099-1102
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Ataluren (PTC124) Induces Cystic Fibrosis Transmembrane Conductance Regulator Protein Expression and Activity in Children with Nonsense Mutation Cystic Fibrosis
Author / Creator:SERMET-GAUDELUS, Isabelle ; DE BOECK, Kris ; MILLERS, Nilsen L ; CONSTANTINE, Scott ; REHA, Allen ; AJAYI, Temitayo ; ELFRING, Gary L ; MILLERS, Langdon L ; CASIMIR, Georges J ; VERMEULEN, François ; LEAL, Teresinha ; MOGENET, Agnès ; ROUSSEL, Delphine ; FRITSCH, Janine ; HANSSENS, Laurence ; HIRAWAT, Samit
Publisher:New York, NY: American Thoracic Society
Journal title:American journal of respiratory and critical care medicine, 2010-11, Vol.182 (10), p.1262-1272
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ArticlesPeer ReviewedFull Text
Correction to: Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015–2022): 2022 interim analy...
Author / Creator:Mercuri, Eugenio ; Osorio, Andrés Nascimento ; Muntoni, Francesco ; Buccella, Filippo ; Desguerre, Isabelle ; Kirschner, Janbernd ; Tulinius, Már ; de Resende, Maria Bernadete Dutra ; Morgenroth, Lauren P. ; Gordish-Dressman, Heather ; Johnson, Shelley ; Kristensen, Allan ; Werner, Christian ; Trifillis, Panayiota ; Henricson, Erik K. ; McDonald, Craig M.
Publisher:Berlin/Heidelberg: Springer Berlin Heidelberg
Journal title:Journal of neurology, 2023-09, Vol.270 (9), p.4583-4583
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Ataluren: First Global Approval

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Ataluren

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Long term treatment with ataluren—the Swedish experience

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Effect of Ataluren on dystrophin mutations

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Ataluren—Promising Therapeutic Premature Termination Codon Readthrough Frontrunner

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Ataluren stimulates ribosomal selection of near-cognate tRNAs to promote nonsense suppression

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Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

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LC–MS/MS quantification of ataluren and ataluren acyl glucuronide in human plasma/urine: application in clinical studies

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Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy

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Proposing a mechanism of action for ataluren

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Ataluren and aminoglycosides stimulate read-through of nonsense codons by orthogonal mechanisms

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Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015–2022): 2022 interim analysis

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Ataluren binds to multiple protein synthesis apparatus sites and competitively inhibits release factor-dependent termination

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Correction of nonsense BMPR2 and SMAD9 mutations by ataluren in pulmonary arterial hypertension

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Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study

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Ataluren suppresses a premature termination codon in an MPS I-H mouse

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Ataluren for the Treatment of Usher Syndrome 2A Caused by Nonsense Mutations

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Ataluren, a New Therapeutic for Alpha-1 Antitrypsin-Deficient Individuals with Nonsense Mutations

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Ataluren (PTC124) Induces Cystic Fibrosis Transmembrane Conductance Regulator Protein Expression and Activity in Children with Nonsense Mutation Cystic Fibrosis

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Correction to: Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015–2022): 2022 interim analy...

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