These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.


BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

222 related articles for article (PubMed ID: 32851872)

  • 1. Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy.
    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; Péréon Y; Renfroe JB; Ryan MM; Sampson JB; Schara U; Sejersen T; Selby K; Tulinius M; Vílchez JJ; Voit T; Wei LJ; Wong BL; Elfring G; Souza M; McIntosh J; Trifillis P; Peltz SW; Muntoni F; ; ;
    J Comp Eff Res; 2020 Oct; 9(14):973-984. PubMed ID: 32851872
    [No Abstract]   [Full Text] [Related]  

  • 2. Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
    McDonald CM; Campbell C; Torricelli RE; Finkel RS; Flanigan KM; Goemans N; Heydemann P; Kaminska A; Kirschner J; Muntoni F; Osorio AN; Schara U; Sejersen T; Shieh PB; Sweeney HL; Topaloglu H; Tulinius M; Vilchez JJ; Voit T; Wong B; Elfring G; Kroger H; Luo X; McIntosh J; Ong T; Riebling P; Souza M; Spiegel RJ; Peltz SW; Mercuri E; ;
    Lancet; 2017 Sep; 390(10101):1489-1498. PubMed ID: 28728956
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy.
    Shieh PB; Elfring G; Trifillis P; Santos C; Peltz SW; Parsons JA; Apkon S; Darras BT; Campbell C; McDonald CM; ; ; ;
    J Comp Eff Res; 2021 Dec; 10(18):1337-1347. PubMed ID: 34693725
    [No Abstract]   [Full Text] [Related]  

  • 4. Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients.
    McDonald CM; Muntoni F; Penematsa V; Jiang J; Kristensen A; Bibbiani F; Goodwin E; Gordish-Dressman H; Morgenroth L; Werner C; Li J; Able R; Trifillis P; Tulinius M;
    J Comp Eff Res; 2022 Feb; 11(3):139-155. PubMed ID: 34791888
    [No Abstract]   [Full Text] [Related]  

  • 5. Ataluren treatment of patients with nonsense mutation dystrophinopathy.
    Bushby K; Finkel R; Wong B; Barohn R; Campbell C; Comi GP; Connolly AM; Day JW; Flanigan KM; Goemans N; Jones KJ; Mercuri E; Quinlivan R; Renfroe JB; Russman B; Ryan MM; Tulinius M; Voit T; Moore SA; Lee Sweeney H; Abresch RT; Coleman KL; Eagle M; Florence J; Gappmaier E; Glanzman AM; Henricson E; Barth J; Elfring GL; Reha A; Spiegel RJ; O'donnell MW; Peltz SW; Mcdonald CM;
    Muscle Nerve; 2014 Oct; 50(4):477-87. PubMed ID: 25042182
    [TBL] [Abstract][Full Text] [Related]  

  • 6. A mini-review and implementation model for using ataluren to treat nonsense mutation Duchenne muscular dystrophy.
    Landfeldt E; Sejersen T; Tulinius M
    Acta Paediatr; 2019 Feb; 108(2):224-230. PubMed ID: 30188594
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study.
    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; ;
    J Comp Eff Res; 2020 Apr; 9(5):341-360. PubMed ID: 31997646
    [No Abstract]   [Full Text] [Related]  

  • 8. The role of ataluren in the treatment of ambulatory and non-ambulatory children with nonsense mutation duchenne muscular dystrophy - a consensus derived using a modified Delphi methodology in Eastern Europe, Greece, Israel and Sweden.
    Golli T; Juříková L; Sejersen T; Dixon C
    BMC Neurol; 2024 Feb; 24(1):73. PubMed ID: 38383326
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Improvements in health status and utility associated with ataluren for the treatment of nonsense mutation Duchenne muscular dystrophy.
    Landfeldt E; Lindberg C; Sejersen T
    Muscle Nerve; 2020 Mar; 61(3):363-368. PubMed ID: 31875971
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy.
    Finkel RS; Flanigan KM; Wong B; Bönnemann C; Sampson J; Sweeney HL; Reha A; Northcutt VJ; Elfring G; Barth J; Peltz SW
    PLoS One; 2013; 8(12):e81302. PubMed ID: 24349052
    [TBL] [Abstract][Full Text] [Related]  

  • 11. European Medicines Agency review of ataluren for the treatment of ambulant patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene.
    Haas M; Vlcek V; Balabanov P; Salmonson T; Bakchine S; Markey G; Weise M; Schlosser-Weber G; Brohmann H; Yerro CP; Mendizabal MR; Stoyanova-Beninska V; Hillege HL
    Neuromuscul Disord; 2015 Jan; 25(1):5-13. PubMed ID: 25497400
    [No Abstract]   [Full Text] [Related]  

  • 12. Continuitiy of care with ataluren in Duchenne Muscular Dystrophy patients with nonsense mutations after loss of ambulation. Personal experience.
    Spagnoli C; Adorisio R; Bello L; D'Amico A; D'Angelo MG; Pane M; Penzo M; Riguzzi P; Sansone V; Vianello A; Fusco C
    Acta Myol; 2023; 42(4):118-122. PubMed ID: 38406379
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Read-through strategies for suppression of nonsense mutations in Duchenne/ Becker muscular dystrophy: aminoglycosides and ataluren (PTC124).
    Finkel RS
    J Child Neurol; 2010 Sep; 25(9):1158-64. PubMed ID: 20519671
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015-2022): 2022 interim analysis.
    Mercuri E; Osorio AN; Muntoni F; Buccella F; Desguerre I; Kirschner J; Tulinius M; de Resende MBD; Morgenroth LP; Gordish-Dressman H; Johnson S; Kristensen A; Werner C; Trifillis P; Henricson EK; McDonald CM;
    J Neurol; 2023 Aug; 270(8):3896-3913. PubMed ID: 37115359
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Early treatment with Ataluren of a 2-year-old boy with nonsense mutation Duchenne dystrophy.
    Bitetti I; Mautone C; Bertella M; Manna MR; Varone A
    Acta Myol; 2021 Dec; 40(4):184-186. PubMed ID: 35047759
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE Registry.
    Muntoni F; Desguerre I; Guglieri M; Osorio AN; Kirschner J; Tulinius M; Buccella F; Elfring G; Werner C; Schilling T; Trifillis P; Zhang O; Delage A; Santos CL; Mercuri E
    J Comp Eff Res; 2019 Oct; 8(14):1187-1200. PubMed ID: 31414621
    [No Abstract]   [Full Text] [Related]  

  • 17. Assessment of face validity of a disease model of nonsense mutation Duchenne muscular dystrophy: a multi-national Delphi panel study.
    Landfeldt E; Zhang R; Childs AM; Johannsen J; O'Rourke D; Sejersen T; Strautmanis J; Schara-Schmidt U; Tulinius M; Walter MC; Willis T; Buesch K
    J Med Econ; 2022; 25(1):808-816. PubMed ID: 35642753
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Good response to the late treatment with ataluren in a boy with Duchenne muscular dystrophy: could the previous mild course of the disease have affected the outcome?
    Pasca L; Gardani A; Paoletti M; Velardo D; Berardinelli A
    Acta Myol; 2022; 41(3):121-125. PubMed ID: 36349184
    [TBL] [Abstract][Full Text] [Related]  

  • 19. The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study.
    McDonald CM; Henricson EK; Abresch RT; Florence JM; Eagle M; Gappmaier E; Glanzman AM; ; Spiegel R; Barth J; Elfring G; Reha A; Peltz S
    Muscle Nerve; 2013 Sep; 48(3):343-56. PubMed ID: 23681930
    [TBL] [Abstract][Full Text] [Related]  

  • 20. The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: reliability, concurrent validity, and minimal clinically important differences from a multicenter study.
    McDonald CM; Henricson EK; Abresch RT; Florence J; Eagle M; Gappmaier E; Glanzman AM; ; Spiegel R; Barth J; Elfring G; Reha A; Peltz SW
    Muscle Nerve; 2013 Sep; 48(3):357-68. PubMed ID: 23674289
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 12.