Safety and Effectiveness of Ataluren: Comparison of Results from the STRIDE Registry and CINRG DMD Natural History Study.
Eugenio Mercuri,Francesco Muntoni,Andres Nascimento Osorio,Mar Tulinius,Filippo Buccella,Lauren P. Morgenroth,Heather Gordish-Dressman,Joel Jiang,Panayiota Trifillis, Lin Zhu,Allan Kristensen,Claudio L. Santos,Erik K. Henricson,Craig M. McDonald,Isabelle Desguerre,G. Bernert,M. Gosk-Tomek,A. Ille,A. Kellersmann, S. Weiss, V Pilshofer,Z. Balintova,P. Danhofer, P. Fabulova, L. Jurikova, P. Fuchsova,J. Haberlova, F. Laffargue,C. Sarret,B. Pontier,R. Bellance, E. Sarrazin,P. Sabouraud,A. Magot, S. Mercier,Y. Pereon,J. M. Cuisset, S. Coopman-Degryse, E. Enaud,M. L. Jacquemont, A. Perville,M. Renouil, V Trommsdorff, D. Verheulpen,S. Fontaine-Carbonnel,C. Vuillerot,S. Peudenier,J. Ropars,F. Audic,B. Chabrol,S. Chabrier, G. Gousse,E. Lagrue, K. Aragon,C. Barnerias, L. Brande,S. De Lucia,I Desguerre,T. Gidaro,A. Seferian,L. Servais,V Laugel,C. Espil-Taris, H. Mecili, E. Raffo, S. Ragot-Mandry, S. Borrell,J. Kirschner,A. Gangfuss, M. Henrich,H. Koelbel,U. Schara, N. Spoenemann, E. Temme, J. Seeger, A. Hirsch,J. Denecke,J. Johannsen,A. Neu, D. Osinski, S. Ruegner, S. Schuessler,R. Trollmann, A. Kaindl, J. B. Schneider, C. Stoltenburg, C. Weiss, G. Schreiber,A. Hahn, M. Grzybowski (Giessen),E. Pavlidou,E. Pavlou,S. Dobner,Z. Liptai,T. Dor,C. Brogna,M. Catteruccia,A. D'Amico,E. Mercuri,M. Pane,L. Bello,E. Pegoraro,C. Semplicini,E. Albamonte,G. Baranello,G. Comi,A. Govoni, A. Lerario,F. Magri,R. Masson,E. Mauri,V Sansone,C. Brusa,T. Mongini,F. Ricci, M. Vacchetti,C. Bruno, C. Paniucci,M. Pedemonte,M. Giannotta,A. Pini,S. Messina,M. Sframeli,G. Vita, L. Ruggiero,L. Santoro,D. Craiu,C. Motoescu, C. Sandu,R. Teleanu, D. Vasile,M. Tulinius,I Hughes,A. M. Childs,Z. Alhaswani,H. Roper,D. Parasuraman, C. DeGoede,V Gowda,A. Manzur,P. Munot, A. Sarkokzy, C. Charlesworth, J. Lemon, L. Turner,S. Spinty,A. Dubrovsky, A. Kornberg,M. Ryan,R. Webster, W. D. Biggar,L. C. McAdam,J. K. Mah,H. Kolski, V. Vishwanathan, S. Chidambaranathan,Y. Nevo,K. Gorni, J. Carlo,C. M. McDonald,E. K. Henricson,R. T. Abresch, N. C. Joyce,A. Cnaan,L. P. Morgenroth,R. Leshner,C. Tesi-Rocha,M. Thangarajh,T. Duong,P. R. Clemens,H. Abdel-Hamid,A. M. Connolly,A. Pestronk, J. Teasley,A. Harper,T. E. Bertorini,N. Kuntz, S. Driscoll,J. W. Day,P. Karachunski,T. Lotze
Journal of Comparative Effectiveness Research（2020）
引用 87|浏览67
关键词
ataluren,dystrophin,effectiveness,nonsense mutation Duchenne muscular dystrophy,safety,STRIDE Registry
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