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

Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy.

Autor: Shieh PB; University of California at Los Angeles, Los Angeles, CA, USA., Elfring G; PTC Therapeutics, Inc., South Plainfield, NJ, USA., Trifillis P; PTC Therapeutics, Inc., South Plainfield, NJ, USA., Santos C; PTC Therapeutics, Inc., South Plainfield, NJ, USA., Peltz SW; PTC Therapeutics, Inc., South Plainfield, NJ, USA., Parsons JA; University of Colorado School of Medicine, Aurora, CO, USA., Apkon S; University of Colorado School of Medicine, Aurora, CO, USA., Darras BT; Boston Children's Hospital, Boston, MA, USA., Campbell C; Children's Hospital - London Health Sciences Centre, University of Western Ontario, London, ON, Canada., McDonald CM; University of California Davis Health, Sacramento, CA, USA.

Publikováno v: Journal of comparative effectiveness research [J Comp Eff Res] 2021 Dec; Vol. 10 (18), pp. 1337-1347. Date of Electronic Publication: 2021 Oct 25.

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

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

Autor: Campbell C; Schulich School of Medicine & Dentistry, Western University, London, ON, N6A 5C1, Canada., Barohn RJ; University of Kansas Medical Center, Kansas City, KS 66160, USA., Bertini E; Bambino Gesù Children's Research Hospital, Rome, 00146, Italy., Chabrol B; Hôpital de la Timone, Unité de Médecine Infantile, Marseille, 13385, France., Comi GP; IRCCS Fondazione Ca'Granda Ospedale Maggiore Policlinico, Dino Ferrari Centre, Department of Pathophysiology & Transplantation, University of Milan, Milan, 20122, Italy., Darras BT; Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA., Finkel RS; Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.; St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Flanigan KM; Nationwide Children's Hospital, Columbus, OH 43205, USA., Goemans N; University Hospitals Leuven, KU Leuven, Leuven, 3000, Belgium., Iannaccone ST; University of Texas Southwestern Medical Center, Dallas, TX 75390, USA., Jones KJ; Kids Neuroscience, The Children's Hospital at Westmead, Westmead, NSW, 2145, Australia., Kirschner J; Department of Neuropediatrics & Muscle Disorders, Medical Center, University of Freiburg, Freiburg 79110, Germany., Mah JK; Department of Pediatrics, Division of Pediatric Neurology, Alberta Children's Hospital, University of Calgary, Calgary, AB T3B 6A8, Canada., Mathews KD; Departments of Pediatrics & Neurology, Division of Pediatric Neurology, University of Iowa Carver College of Medicine, Iowa City, IA 52242, USA., McDonald CM; University of California Davis Health, Sacramento, CA 95817, USA., Mercuri E; Department of Pediatric Neurology, Catholic University, Rome, 00168, Italy., Nevo Y; Schneider Children's Medical Center, Tel Aviv University, Tel Aviv, 6997801, Israel., Péréon Y; Reference Centre for Neuromuscular Disorders AOC, Hôtel-Dieu, Nantes, 44000, France., Renfroe JB; Child Neurology Center of Northwest Florida, Gulf Breeze, FL 32561, USA., Ryan MM; The Royal Children's Hospital, Parkville, Victoria, 3052, Australia., Sampson JB; Stanford University Medical Center, Department of Neurology & Neurological Sciences, Stanford, CA 94305, USA., Schara U; Department of Pediatric Neurology, University Hospital Essen, University of Duisburg-Essen, Essen, 45122, Germany., Sejersen T; Karolinska University Hospital, Karolinska Institutet, Stockholm, 171 76, Sweden., Selby K; Division of Neurology, British Columbia Children's Hospital, Vancouver, BC, V6H 3N1, Canada., Tulinius M; Gothenburg University, Queen Silvia Children's Hospital, Gothenburg, 416 85, Sweden., Vílchez JJ; Hospital Universitario y Politécnico La Fe, CIBERER, Valencia, 46026, Spain., Voit T; NIHR Great Ormond Street Hospital Biomedical Research Centre, University College London & UCL Great Ormond Street Institute of Child Health, Great Ormond Street Hospital Trust, London, WC1N 1EH, UK., Wei LJ; Harvard TH Chan School of Public Health, Harvard University, Boston, MA 02115, USA., Wong BL; University of Massachusetts Medical School, UMass, Worcester, MA 01655, USA., Elfring G; PTC Therapeutics Inc., South Plainfield, NJ 07080, USA., Souza M; PTC Therapeutics Inc., South Plainfield, NJ 07080, USA., McIntosh J; PTC Therapeutics Inc., South Plainfield, NJ 07080, USA., Trifillis P; PTC Therapeutics Inc., South Plainfield, NJ 07080, USA., Peltz SW; PTC Therapeutics Inc., South Plainfield, NJ 07080, USA., Muntoni F; NIHR Great Ormond Street Hospital Biomedical Research Centre, University College London & UCL Great Ormond Street Institute of Child Health, Great Ormond Street Hospital Trust, London, WC1N 1EH, UK.

Publikováno v: Journal of comparative effectiveness research [J Comp Eff Res] 2020 Oct; Vol. 9 (14), pp. 973-984. Date of Electronic Publication: 2020 Aug 27.