BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

174 related articles for article (PubMed ID: 38383326)

  • 1. 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]  

  • 2. 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]  

  • 3. 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]  

  • 4. 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]  

  • 5. 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]  

  • 6. 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]  

  • 7. 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]  

  • 8. 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]  

  • 9. 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]  

  • 10. Off-Label Use of Ataluren in Four Non-ambulatory Patients With Nonsense Mutation Duchenne Muscular Dystrophy: Effects on Cardiac and Pulmonary Function and Muscle Strength.
    Ebrahimi-Fakhari D; Dillmann U; Flotats-Bastardas M; Poryo M; Abdul-Khaliq H; Shamdeen MG; Mischo B; Zemlin M; Meyer S
    Front Pediatr; 2018; 6():316. PubMed ID: 30406066
    [TBL] [Abstract][Full Text] [Related]  

  • 11. 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]  

  • 12. 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]  

  • 13. 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]  

  • 14. 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]  

  • 15. Long term treatment with ataluren-the Swedish experience.
    Michael E; Sofou K; Wahlgren L; Kroksmark AK; Tulinius M
    BMC Musculoskelet Disord; 2021 Sep; 22(1):837. PubMed ID: 34592975
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Treatment with ataluren in four symptomatic Duchenne carriers. A pilot study.
    Dori A; Scutifero M; Passamano L; Zoppi D; Ruggiero L; Trabacca A; Politano L
    Acta Myol; 2024; 43(1):8-15. PubMed ID: 38586166
    [TBL] [Abstract][Full Text] [Related]  

  • 17. 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]  

  • 18. 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]  

  • 19. Therapeutic approach with Ataluren in Duchenne symptomatic carriers with nonsense mutations in dystrophin gene. Results of a 9-month follow-up in a case report.
    D'Ambrosio P; Orsini C; Nigro V; Politano L
    Acta Myol; 2018 Dec; 37(4):272-274. PubMed ID: 30944907
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Can symptomatic nmDuchenne carriers benefit from treatment with ataluren? Results of 193-month follow-up.
    Dori A; Guglieri M; Scutifero M; Passamano L; Trabacca A; Politano L
    Acta Myol; 2021 Dec; 40(4):152-157. PubMed ID: 35047755
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 9.