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ArticlesPeer ReviewedFull Text
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
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
Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy
Author / Creator:Campbell, Craig ; Barohn, Richard J ; Bertini, Enrico ; Chabrol, Brigitte ; Comi, Giacomo Pietro ; Darras, Basil T ; Finkel, Richard S ; Flanigan, Kevin M ; Goemans, Nathalie ; Iannaccone, Susan T ; Jones, Kristi J ; Kirschner, Janbernd ; Mah, Jean K ; Mathews, Katherine D ; McDonald, Craig M ; Mercuri, Eugenio ; Nevo, Yoram ; Péréon, Yann ; Renfroe, J Ben ; Ryan, Monique M ; Sampson, Jacinda B ; Schara, Ulrike ; Sejersen, Thomas ; Selby, Kathryn ; Tulinius, Már ; Vílchez, Juan J ; Voit, Thomas ; Wei, Lee-Jen ; Wong, Brenda L ; Elfring, Gary ; Souza, Marcio ; McIntosh, Joseph ; Trifillis, Panayiota ; Peltz, Stuart W ; Muntoni, Francesco
Publisher:England: Future Medicine Ltd
Journal title:Journal of comparative effectiveness research, 2020-10, Vol.9 (14), p.973-984
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ArticlesPeer ReviewedFull Text
Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE Registry
Author / Creator:Muntoni, Francesco ; Desguerre, Isabelle ; Guglieri, Michela ; Osorio, Andrés Nascimento ; Kirschner, Janbernd ; Tulinius, Már ; Buccella, Filippo ; Elfring, Gary ; Werner, Christian ; Schilling, Traci ; Trifillis, Panayiota ; Zhang, Olivia ; Delage, Abdallah ; Santos, Claudio L ; Mercuri, Eugenio
Publisher:England: Future Medicine Ltd
Journal title:Journal of comparative effectiveness research, 2019-10, Vol.8 (14), p.1187-1200
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The role of ataluren in the treatment of ambulatory and non-ambulatory children with nonsense mutation duchenne muscular dystrophy - a consensus derived using a modified Delphi methodology in Eastern...
Author / Creator:Golli, Tanja ; Juříková, Lenka ; Sejersen, Thomas ; Dixon, Craig
Publisher:England: BioMed Central Ltd
Journal title:BMC neurology, 2024-02, Vol.24 (1), p.73-73, Article 73
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A qualitative study on the impact of caring for an ambulatory individual with nonsense mutation Duchenne muscular dystrophy
Author / Creator:Williams, Kate ; Davidson, Ian ; Rance, Mark ; Buesch, Katharina ; Acaster, Sarah
Publisher:Cham: Springer International Publishing
Journal title:Journal of patient-reported outcomes, 2021-08, Vol.5 (1), p.71-71, Article 71
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Symptoms and impacts of ambulatory nonsense mutation Duchenne muscular dystrophy: a qualitative study and the development of a patient-centred conceptual model
Author / Creator:Williams, Kate ; Davidson, Ian ; Rance, Mark ; Boehnke, Axel ; Buesch, Katharina ; Acaster, Sarah
Publisher:Cham: Springer International Publishing
Journal title:Journal of patient-reported outcomes, 2021-08, Vol.5 (1), p.75-75, Article 75
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ArticlesPeer ReviewedFull Text
A.06 Ataluren: an overview of clinical trial results in nonsense mutation Duchenne Muscular Dystrophy (nmDMD)
Author / Creator:McDonald, CM ; Bushby, K ; Tulinius, M ; Finkel, R ; Topaloglu, H ; Day, JW ; Flanigan, K ; Lowes, L ; Eagle, M ; Luo, X ; Elfring, G ; Kroger, H ; Riebling, P ; Ong, T ; Spiegel, R ; Peltz, SW
Publisher:New York, USA: Cambridge University Press
Journal title:Canadian journal of neurological sciences, 2016-06, Vol.43 (S2), p.S8-S8
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ArticlesPeer ReviewedFull Text
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
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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ArticlesPeer ReviewedFull Text
Phase 2a Study of Ataluren-Mediated Dystrophin Production in Patients with Nonsense Mutation Duchenne Muscular Dystrophy: e81302
Author / Creator:Finkel, Richard S ; Flanigan, Kevin M ; Wong, Brenda ; Bonnemann, Carsten ; Sampson, Jacinda ; Sweeney, H Lee ; Reha, Allen ; Northcutt, Valerie J ; Elfring, Gary ; Barth, Jay
Journal title:PloS one, 2013-12, Vol.8 (12)
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Newsletter articlesFull Text
PTC Therapeutics Says CHMP Of European Medicines Agency Maintained Its Negative Opinion On Renewal Of Conditional Marketing Authorization Of Translarna For Treatment Of Nonsense Mutation Duchenne Musc...
Publisher:Southfield: Accretive Capital LLC d/b/a Benzinga
Journal title:Benzinga Newswires, 2024
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ArticlesFull Text
New Findings from Western University in Duchenne Muscular Dystrophy Provides New Insights (Meta-analyses of Ataluren Randomized Controlled Trials In Nonsense Mutation Duchenne Muscular Dystrophy)
Publisher:NewsRX LLC
Journal title:Obesity, fitness, & wellness week, 2021-02, p.3509
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Newsletter articlesFull Text
PTC Therapeutics to Host Call to Review Topline Results from Study 041 for Translarna™ (ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy
Publisher:New York: PR Newswire Association LLC
Journal title:PR Newswire, 2022
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Newsletter articlesFull Text
Press Release: PTC Therapeutics to Host Call to Review Topline Results from Study 041 for Translarna(TM) (ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy
Publisher:New York: Dow Jones & Company Inc
Journal title:Dow Jones Institutional News, 2022
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Newsletter articlesFull Text
STRIDE Data Show Translarna™ Delays Loss of Ambulation by More Than Five Years in Boys with Nonsense Mutation Duchenne Muscular Dystrophy: Translarna was seen to slow disease progression compared to s...
Publisher:New York: PR Newswire Association LLC
Journal title:PR Newswire, 2021
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Newsletter articlesFull Text
United States : STRIDE Data Show Translarna Delays Loss of Ambulation by More Than Five Years in Boys with Nonsense Mutation Duchenne Muscular Dystrophy
Publisher:Camden: Disco Digital Media, Inc
Journal title:MENA Report, 2021
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Newsletter articlesFull Text
Press Release: STRIDE Data Show Translarna(TM) Delays Loss of Ambulation by More Than Five Years in Boys with Nonsense Mutation Duchenne Muscular Dystrophy
Publisher:New York: Dow Jones & Company Inc
Journal title:Dow Jones Institutional News, 2021
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Newsletter articlesFull Text
United States : PTC Therapeutics to Host Call to Review Results from its Study of Translarna (ataluren) in Patients with Nonsense Mutation Duchenne Muscular Dystrophy
Publisher:Camden: Disco Digital Media, Inc
Journal title:MENA Report, 2021
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Newsletter articlesFull Text
PTC Therapeutics to Host Call to Review Results from its Study of Translarna™ (ataluren) in Patients with Nonsense Mutation Duchenne Muscular Dystrophy
Publisher:New York: PR Newswire Association LLC
Journal title:PR Newswire, 2021
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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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Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy

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Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy

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Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE Registry

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The role of ataluren in the treatment of ambulatory and non-ambulatory children with nonsense mutation duchenne muscular dystrophy - a consensus derived using a modified Delphi methodology in Eastern...

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A qualitative study on the impact of caring for an ambulatory individual with nonsense mutation Duchenne muscular dystrophy

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Symptoms and impacts of ambulatory nonsense mutation Duchenne muscular dystrophy: a qualitative study and the development of a patient-centred conceptual model

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A.06 Ataluren: an overview of clinical trial results in nonsense mutation Duchenne Muscular Dystrophy (nmDMD)

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

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Phase 2a Study of Ataluren-Mediated Dystrophin Production in Patients with Nonsense Mutation Duchenne Muscular Dystrophy: e81302

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PTC Therapeutics Says CHMP Of European Medicines Agency Maintained Its Negative Opinion On Renewal Of Conditional Marketing Authorization Of Translarna For Treatment Of Nonsense Mutation Duchenne Musc...

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New Findings from Western University in Duchenne Muscular Dystrophy Provides New Insights (Meta-analyses of Ataluren Randomized Controlled Trials In Nonsense Mutation Duchenne Muscular Dystrophy)

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PTC Therapeutics to Host Call to Review Topline Results from Study 041 for Translarna™ (ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy

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Press Release: PTC Therapeutics to Host Call to Review Topline Results from Study 041 for Translarna(TM) (ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy

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STRIDE Data Show Translarna™ Delays Loss of Ambulation by More Than Five Years in Boys with Nonsense Mutation Duchenne Muscular Dystrophy: Translarna was seen to slow disease progression compared to s...

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United States : STRIDE Data Show Translarna Delays Loss of Ambulation by More Than Five Years in Boys with Nonsense Mutation Duchenne Muscular Dystrophy

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Press Release: STRIDE Data Show Translarna(TM) Delays Loss of Ambulation by More Than Five Years in Boys with Nonsense Mutation Duchenne Muscular Dystrophy

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United States : PTC Therapeutics to Host Call to Review Results from its Study of Translarna (ataluren) in Patients with Nonsense Mutation Duchenne Muscular Dystrophy

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PTC Therapeutics to Host Call to Review Results from its Study of Translarna™ (ataluren) in Patients with Nonsense Mutation Duchenne Muscular Dystrophy

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