Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: A meta‐analysis of disease progression rates in recent multicenter clinical trials

Autor: McDonald CM, A238., Sajeev, G, Yao, Z, Mcdonnell, E, Elfring, G, Souza, M, Peltz, Sw, Darras, Bt, Shieh, Pb, Cox, Da, Landry, J, Signorovitch, J, Campbell, C, Torricelli, Re, Finkel, Rs, Flanigan, Km, Goemans, N, Heydemann, P, Kaminska, A, Kirschner, J, Muntoni, F, Osorio, An, Schara, U, Sejersen, T, Sweeney, Hl, Topaloglu, H, Tulinius, M, Vilchez, Jj, Voit, T, Wong, B, Alfano, Ln, Eagle, M, James, Mk, Lowes, L, Mayhew, A, Mazzone, Es, Nelson, L, Rose, Kj, Abdel-Hamid, Hz, Apkon, Sd, Barohn, Rj, Bertini, E, Bloetzer, C, de Vaud LC, Butterfield, Rj, Chabrol, B, Chae, Jh, Jongno-Gu, Dr, Comi, Gp, Dastgir, J, Desguerre, I, Escobar, Rg, Finanger, E, Guglieri, M, Hughes, I, Iannaccone, St, Jones, Kj, Karachunski, P, Kudr, M, Lotze, T, Mah, Jk, Mathews, K, Nevo, Y, Parsons, J, Péréon, Y, de Queiroz Campos Araujo AP, Renfroe, Jb, de Mbd, R, Ryan, M, Selby, K, Tennekoon, G, Vita, G, Abdel-Hamid, H, Apkon, S, Barohn, R, Belousova, E, Brandsema, J, Bruno, C, Burnette, W, Butterfield, R, Byrne, B, Carlo, J, Chandratre, S, Comi, G, Connolly, A, De Groot, I, Deconinck, N, Dooley, J, Dubrovsky, A, Durigneux, J, Finkel, R, Frank, Lm, Harper, A, Hattori, A, Herguner, O, Iannaccone, S, Janas, J, Jong, Yj, Komaki, H, Kuntz, N, Lee, Wt, Leung, E, Mah, J, Cm, M, Mercuri, E, Mcmillan, H, Mueller-Felber, W, Lopez de Munain, A, Nakamura, A, Niks, E, Ogata, K, Pascual, S, Pegoraro, E, Pereon, Y, Renfroe, B, Sanka, Rb, Schallner, J, Sendra, Ii, Servais, L, Smith, E, Sparks, S, Victor, R, Wicklund, M, Wilichoswki, E, Wong, B.

Publikováno v: Muscle & Nerve

Introduction In this study we characterized disease progression over 48 weeks among boys receiving deflazacort vs prednisone/prednisolone placebo arm treatment in two recent Duchenne muscular dystrophy (DMD) clinical trials. Methods Ambulatory boys w

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::226b242305b42902003713d1980f97f5
https://doi.org/10.1002/mus.26736

Group Sequential Designs for Clinical Trials

Autor: Elfring, G. L., Schultz, J. R.

Publikováno v: Biometrics, 1973 Sep 01. 29(3), 471-477.

Externí odkaz: https://www.jstor.org/stable/2529170

Atalauren: an overview of clinical trial results in nonsense mutation Duchenne muscular dystrophy

Autor: Comi, G. P., Bertini, E., Mercuri, E., Messina, Sonia, Lou, X., Elfring, G., Kroger, H., Riebling, P., Ong, T., Spiegel, R., Peltz, S., The Atalauren DMD Study Group

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=od______3978::4a1b3f51949c906e17976a9fa2998e64
http://hdl.handle.net/11570/3095935

DMD CLINICAL THERAPIES I: P.112STRIDE™: A patient registry study examining the use of ataluren (Translarna™) in patients with nonsense mutation muscular dystrophy (nmDMD)

Autor: Muntoni, F., Desguerre, I., Guglieri, M., Mercuri, E., Nascimento Osorio, A., Kirschner, J., Tulinius, M., Buccello, F., Delage, A., Elfring, G., Werner, C., Schilling, T., Trifillis, P.

Publikováno v: In Neuromuscular Disorders October 2018 28 Supplement 2:S63-S63

D20 - Meta-analyses of ataluren in patients with nonsense mutation Duchenne muscular dystrophy

Autor: Muntoni, F., Mercuri, E., Luo, X., Elfring, G., Werner, C., Trifillis, P., Peltz, S.W., McDonald, C.M.

Publikováno v: In Neuromuscular Disorders April 2018 28 Supplement 1:S12-S12