Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial von McDonald, Craig M., Shieh, Perry B., Abdel-Hamid, Hoda Z., Connolly, Anne M., Ciafaloni, Emma, Wagner, Kathryn R., Goemans, Nathalie, Mercuri, Eugenio, Khan, Navid, Koenig, Erica, Malhotra, Jyoti, Zhang, Wenfei, Han, Baoguang, Mendell, Jerry R.

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

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Major Adverse Dystrophinopathy Events (MADE) score as marker of cumulative morbidity and risk for mortality in boys with Duchenne muscular dystrophy von Kaufman, Beth D., Garcia, Ariadna, He, Zihuai, Tesi-Rocha, Carolina, Buu, MyMy, Rosenthal, David, Gordish-Dressman, Heather, Almond, Christopher S., Duong, Tina

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Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study von Mercuri, E, Muntoni, F, Osorio, AN, Tulinius, M, Buccella, F, Morgenroth, LP, Gordish-Dressman, H, Jiang, J, Trifillis, P, Zhu, J, Kristensen, A, Santos, CL, Henricson, EK, McDonald, CM, Desguerre, I, STRIDE, CINRG Duchenne Natural History Investigators

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Supplementary figures: Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients von McDonald, Craig M, Muntoni, Francesco, Penematsa, Vinay, Jiang, Joel, Kristensen, Allan, Bibbiani, Francesco, Goodwin, Elizabeth, Gordish-Dressman, Heather, Morgenroth, Lauren, Werner, Christian, Li, James, Able, Richard, Trifillis, Panayiota, Tulinius, Már, Investigators, On behalf of the Study 019 & CINRG Duchenne Natural History

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Supplementary tables: Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients von McDonald, Craig M, Muntoni, Francesco, Penematsa, Vinay, Jiang, Joel, Kristensen, Allan, Bibbiani, Francesco, Goodwin, Elizabeth, Gordish-Dressman, Heather, Morgenroth, Lauren, Werner, Christian, Li, James, Able, Richard, Trifillis, Panayiota, Tulinius, Már, Investigators, On behalf of the Study 019 & CINRG Duchenne Natural History

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Supplementary materials: Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients.docx von McDonald, Craig, Muntoni, Francesco, Penematsa, Vinay, Jiang, Joel, Kristensen, Allan, Bibbiani, Francesco, Goodwin, Elizabeth, Gordish-Dressman, Heather, Morgenroth, Lauren, Werner, Christian, Li, James, Able, Richard, Trifillis, Panayiota, Tulinius, Már, Investigators, On behalf of the Study 019 & CINRG Duchenne Natural History

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Supplementary Figure 2: Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients.pdf von McDonald, Craig, Muntoni, Francesco, Penematsa, Vinay, Jiang, Joel, Kristensen, Allan, Bibbiani, Francesco, Goodwin, Elizabeth, Gordish-Dressman, Heather, Morgenroth, Lauren, Werner, Christian, Li, James, Able, Richard, Trifillis, Panayiota, Tulinius, Már, Investigators, On behalf of the Study 019 & CINRG Duchenne Natural History

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Supplementary Figure 1: Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients.pdf von McDonald, Craig, Muntoni, Francesco, Penematsa, Vinay, Jiang, Joel, Kristensen, Allan, Bibbiani, Francesco, Goodwin, Elizabeth, Gordish-Dressman, Heather, Morgenroth, Lauren, Werner, Christian, Li, James, Able, Richard, Trifillis, Panayiota, Tulinius, Már, Investigators, On behalf of the Study 019 & CINRG Duchenne Natural History

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Plain Language Summary: Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients.pdf von McDonald, Craig, Muntoni, Francesco, Penematsa, Vinay, Jiang, Joel, Kristensen, Allan, Bibbiani, Francesco, Goodwin, Elizabeth, Gordish-Dressman, Heather, Morgenroth, Lauren, Werner, Christian, Li, James, Able, Richard, Trifillis, Panayiota, Tulinius, Már, Investigators, On behalf of the Study 019 & CINRG Duchenne Natural History

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A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment von Mitelman, Olga, Abdel-Hamid, Hoda Z., Byrne, Barry J., Connolly, Anne M., Heydemann, Peter, Proud, Crystal, Shieh, Perry B., Wagner, Kathryn R., Dugar, Ashish, Santra, Sourav, Signorovitch, James, Goemans, Nathalie, McDonald, Craig M., Mercuri, Eugenio, Mendell, Jerry R.

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