Zobrazeno 1 - 10
of 2 466
pro vyhledávání: '"Iannaccone, ST"'
Autor:
Iannaccone ST; Departments of Pediatrics and Neurology, University of Texas Southwestern Medical Center and Children's Health, Dallas, TX, USA. Susan.Iannaccone@UTSouthwestern.edu., Cai C; Department of Pathology, University of Texas Southwestern Medical Center and Children's Health, Dallas, TX, USA. Chunyu.Cai@UTSouthwestern.edu., Rhem B; Departments of Pediatrics and Neurology, University of Texas Southwestern Medical Center and Children's Health, Dallas, TX, USA., Batley K; Departments of Pediatrics and Neurology, University of Texas Southwestern Medical Center and Children's Health, Dallas, TX, USA., Rajaram V; Department of Pathology, University of Texas Southwestern Medical Center and Children's Health, Dallas, TX, USA., Greenberg BM; Department of Neurology, University of Texas Southwestern Medical Center and Children's Health, Dallas, TX, USA., Dharia S; Sarepta Therapeutics, Inc., Cambridge, MA, USA., Zaidman CM; Department of Neurology, Washington University, St. Louis, MO, USA.
Publikováno v:
Journal of neurology [J Neurol] 2024 Aug; Vol. 271 (8), pp. 5659-5664. Date of Electronic Publication: 2024 Jun 22.
Autor:
Pineda ED; Genentech, Inc., 1 DNA Way, South San Francisco, CA, 94080, USA. pineda.elmor@gene.com., To TM; Genentech, Inc., 1 DNA Way, South San Francisco, CA, 94080, USA., Dickendesher TL; Genentech, Inc., 1 DNA Way, South San Francisco, CA, 94080, USA., Shapouri S; Genentech, Inc., 1 DNA Way, South San Francisco, CA, 94080, USA., Iannaccone ST; Departments of Pediatrics and Neurology, UT Southwestern Medical Center, 5323 Harry Hines Blvd, Dallas, TX, USA.
Publikováno v:
Advances in therapy [Adv Ther] 2024 Jun; Vol. 41 (6), pp. 2446-2459. Date of Electronic Publication: 2024 May 06.
Observing the Clinical Course of Duchenne Muscular Dystrophy in Medicaid Real-World Healthcare Data.
Autor:
Qian C; Broadstreet Health Economics and Outcomes Research, 201-343 Railway Street, Vancouver, BC, V6A 1A4, Canada., Klimchak AC; Sarepta Therapeutics, Inc, 215 First Street, Cambridge, MA, 02142, USA., Szabo SM; Broadstreet Health Economics and Outcomes Research, 201-343 Railway Street, Vancouver, BC, V6A 1A4, Canada. sszabo@broadstreetheor.com., Popoff E; Broadstreet Health Economics and Outcomes Research, 201-343 Railway Street, Vancouver, BC, V6A 1A4, Canada., Iannaccone ST; University of Texas Southwestern, 5323 Harry Hines Blvd, Dallas, TX, 75390, USA., Gooch KL; Sarepta Therapeutics, Inc, 215 First Street, Cambridge, MA, 02142, USA.
Publikováno v:
Advances in therapy [Adv Ther] 2024 Jun; Vol. 41 (6), pp. 2519-2530. Date of Electronic Publication: 2024 May 02.
Publikováno v:
American Journal of Medical Genetics. Part A; September 2008, Vol. 146 Issue: 18 p2318-2326, 9p
Autor:
Audhya IF; Sarepta Therapeutics, Inc., Cambridge, MA, USA., Szabo SM; Broadstreet HEOR, 201-343 Railway St, Vancouver, BC, V6A 1A4, Canada. sszabo@broadstreetheor.com., Bever A; Broadstreet HEOR, 201-343 Railway St, Vancouver, BC, V6A 1A4, Canada., O'Sullivan F; Broadstreet HEOR, 201-343 Railway St, Vancouver, BC, V6A 1A4, Canada., Malone DC; The University of Utah, Salt Lake City, UT, USA., Feeny D; McMaster University, Hamilton, ON, Canada., Neumann P; Tufts Medical Center, Boston, MA, USA., Iannaccone ST; The University of Texas Southwestern, Dallas, TX, USA., Jayasinghe P; Broadstreet HEOR, 201-343 Railway St, Vancouver, BC, V6A 1A4, Canada., Gooch KL; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
Publikováno v:
Journal of patient-reported outcomes [J Patient Rep Outcomes] 2023 Dec 15; Vol. 7 (1), pp. 132. Date of Electronic Publication: 2023 Dec 15.
Autor:
Fox D; Genentech Inc, 1 DNA Way, South San Francisco, CA, USA. fox.david@gene.com., To TM; Genentech Inc, 1 DNA Way, South San Francisco, CA, USA., Seetasith A; Genentech Inc, 1 DNA Way, South San Francisco, CA, USA., Patel AM; Genentech Inc, 1 DNA Way, South San Francisco, CA, USA., Iannaccone ST; Departments of Pediatrics and Neurology, University of Texas Southwestern Medical Center, Dallas, TX, USA.
Publikováno v:
Advances in therapy [Adv Ther] 2023 Mar; Vol. 40 (3), pp. 903-919. Date of Electronic Publication: 2022 Dec 19.
Autor:
Szabo, S.M., Audhya, IF, Bever, A, Sun, R, Griffin, E, Feeny, D, Malone, D, Neumann, P, Iannaccone, ST, Gooch, KL
Publikováno v:
In Value in Health June 2024 27(6) Supplement:S303-S303
Autor:
Aguayo JS; Department of Pediatrics, Division of Hematology/Oncology, University of Texas Southwestern Medical Center, Dallas, TX, USA., Shelton JM; Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas, TX, USA., Tan W; Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center, University of Texas Southwestern Medical Center, Dallas, TX, USA., Rakheja D; Department of Pathology, University of Texas Southwestern Medical Center, Dallas, TX, USA., Cai C; Department of Pathology, University of Texas Southwestern Medical Center, Dallas, TX, USA., Shalaby A; Lyda Hill Department of Bioinformatics, University of Texas Southwestern Medical Center, Dallas, TX, USA., Lee J; Lyda Hill Department of Bioinformatics, University of Texas Southwestern Medical Center, Dallas, TX, USA., Iannaccone ST; Departments of Pediatrics and Neurology, University of Texas Southwestern Medical Center, Dallas, TX, USA., Xu L; Department of Pediatrics, Division of Hematology/Oncology, University of Texas Southwestern Medical Center, Dallas, TX, USA; Quantitative Biomedical Research Center, Peter O'Donnell Jr. School of Public Health, University of Texas Southwestern Medical Center, Dallas, TX, USA., Chen K; Department of Pediatrics, Division of Hematology/Oncology, University of Texas Southwestern Medical Center, Dallas, TX, USA; Harold C. Simmons Comprehensive Cancer Center, University of Texas Southwestern Medical Center, Dallas, TX, USA; Gill Center for Cancer and Blood Disorders, Children's Health Children's Medical Center, Dallas, TX, USA., Burns DK; Department of Pathology, University of Texas Southwestern Medical Center, Dallas, TX, USA., Zheng Y; Department of Pediatrics, Division of Hematology/Oncology, University of Texas Southwestern Medical Center, Dallas, TX, USA; Harold C. Simmons Comprehensive Cancer Center, University of Texas Southwestern Medical Center, Dallas, TX, USA. Electronic address: Yanbin.Zheng@UTSouthwestern.edu.
Publikováno v:
Biochemical and biophysical research communications [Biochem Biophys Res Commun] 2023 Jul 12; Vol. 665, pp. 159-168. Date of Electronic Publication: 2023 May 03.
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
https://doi.org/10.1002/mus.26736
Autor:
Campbell, C, Barohn, RJ, Bertini, E, Chabrol, B, Comi, GP, Darras, BT, Finkel, RS, Flanigan, KM, Goemans, N, Iannaccone, ST, Jones, KJ, Kirschner, J, Mah, JK, Mathews, KD, McDonald, CM, Mercuri, E, Nevo, Y, Pereon, Y, Ben Renfroe, J, Ryan, MM, Sampson, JB, Schara, U, Sejersen, T, Selby, K, Tulinius, M, VILCHEZ, JJ, Voit, T, Wei, LJ, Wong, BL, Elfring, G, Souza, M, McIntosh, J, Trifillis, P, Peltz, SW, Muntoni, F, PTC124-GD-007-Study Grp, ACT DMD Study Grp, Clinical Evaluator Training Grp
Publikováno v:
Journal of Comparative Effectiveness Research
r-IIS La Fe. Repositorio Institucional de Producción Científica del Instituto de Investigación Sanitaria La Fe
instname
r-IIS La Fe. Repositorio Institucional de Producción Científica del Instituto de Investigación Sanitaria La Fe
instname
Aim:Assess the totality of efficacy evidence for ataluren in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD).Materials & methods:Data from the two completed randomized controlled trials (ClinicalTrials.gov: NCT00592553; NCT0182648
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=RECOLECTA___::edd110e20c23be981bc90e66cf3ae59c
https://fundanet.iislafe.san.gva.es/publicaciones/ProdCientif/PublicacionFrw.aspx?id=12630
https://fundanet.iislafe.san.gva.es/publicaciones/ProdCientif/PublicacionFrw.aspx?id=12630
