180 related articles for article (PubMed ID: 31414621)
1. 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]
2. 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]
3. 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]
4. 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]
5. 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]
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. 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]
8. 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]
9. 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]
10. 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]
11. 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]
12. 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]
13. 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]
14. [Expert recommendation: treatment of nonambulatory patients with Duchenne muscular dystrophy].
Bernert G; Hahn A; Köhler C; Meyer S; Schara U; Schlachter K; Trollmann R; Walter MC
Nervenarzt; 2021 Apr; 92(4):359-366. PubMed ID: 33215271
[TBL] [Abstract][Full Text] [Related]
15. 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]
16. 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]
17. Ataluren: first global approval.
Ryan NJ
Drugs; 2014 Sep; 74(14):1709-14. PubMed ID: 25193627
[TBL] [Abstract][Full Text] [Related]
18. 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]
19. Clinical and mutational characteristics of Duchenne muscular dystrophy patients based on a comprehensive database in South China.
Wang DN; Wang ZQ; Yan L; He J; Lin MT; Chen WJ; Wang N
Neuromuscul Disord; 2017 Aug; 27(8):715-722. PubMed ID: 28318817
[TBL] [Abstract][Full Text] [Related]
20. 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]
[Next] [New Search]