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2. 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]
3. Reading through premature stop codons with PTC1 24. Project Catalyst to find more Duchenne drugs. Interview by Guenter Scheuerbrandt. Welch E; Goetz D; Almstead N Acta Myol; 2008 Oct; 27():63-8. PubMed ID: 19364064 [No Abstract] [Full Text] [Related]
5. 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]
6. Studies point way to new therapeutic prospects for muscular dystrophy. Kuehn BM JAMA; 2007 Sep; 298(12):1385-6. PubMed ID: 17895449 [No 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. 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. Aminoglycoside treatment for muscular dystrophy is scientifically rational, but is it clinically effective? Hirano M Curr Neurol Neurosci Rep; 2002 Jan; 2(1):53-4. PubMed ID: 11898583 [No Abstract] [Full Text] [Related]
12. Gentamicin treatment of Duchenne and Becker muscular dystrophy due to nonsense mutations. Wagner KR; Hamed S; Hadley DW; Gropman AL; Burstein AH; Escolar DM; Hoffman EP; Fischbeck KH Ann Neurol; 2001 Jun; 49(6):706-11. PubMed ID: 11409421 [TBL] [Abstract][Full Text] [Related]
13. Letter to the Editor. Scheuerbrandt G Acta Myol; 2015 May; 34(1):14. PubMed ID: 26155065 [No Abstract] [Full Text] [Related]
14. Sequence specificity of aminoglycoside-induced stop condon readthrough: potential implications for treatment of Duchenne muscular dystrophy. Howard MT; Shirts BH; Petros LM; Flanigan KM; Gesteland RF; Atkins JF Ann Neurol; 2000 Aug; 48(2):164-9. PubMed ID: 10939566 [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]
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18. Genetics and emerging treatments for Duchenne and Becker muscular dystrophy. Wein N; Alfano L; Flanigan KM Pediatr Clin North Am; 2015 Jun; 62(3):723-42. PubMed ID: 26022172 [TBL] [Abstract][Full Text] [Related]
19. Assessment of Treatment Effect With Multiple Outcomes in 2 Clinical Trials of Patients With Duchenne Muscular Dystrophy. Li D; McDonald CM; Elfring GL; Souza M; McIntosh J; Kim DH; Wei LJ JAMA Netw Open; 2020 Feb; 3(2):e1921306. PubMed ID: 32058550 [No Abstract] [Full Text] [Related]
20. 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] [Next] [New Search]