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23. Quantitative Antisense Screening and Optimization for Exon 51 Skipping in Duchenne Muscular Dystrophy. Echigoya Y; Lim KRQ; Trieu N; Bao B; Miskew Nichols B; Vila MC; Novak JS; Hara Y; Lee J; Touznik A; Mamchaoui K; Aoki Y; Takeda S; Nagaraju K; Mouly V; Maruyama R; Duddy W; Yokota T Mol Ther; 2017 Nov; 25(11):2561-2572. PubMed ID: 28865998 [TBL] [Abstract][Full Text] [Related]
24. Exon skipping therapy for Duchenne muscular dystrophy. Kole R; Krieg AM Adv Drug Deliv Rev; 2015 Jun; 87():104-7. PubMed ID: 25980936 [TBL] [Abstract][Full Text] [Related]
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32. Antisense oligonucleotide drugs for Duchenne muscular dystrophy: how far have we come and what does the future hold? Guncay A; Yokota T Future Med Chem; 2015; 7(13):1631-5. PubMed ID: 26423833 [No Abstract] [Full Text] [Related]
38. Approving a Problematic Muscular Dystrophy Drug: Implications for FDA Policy. Kesselheim AS; Avorn J JAMA; 2016 Dec; 316(22):2357-2358. PubMed ID: 27775756 [No Abstract] [Full Text] [Related]
39. Delayed onset of ambulation in boys with Duchenne muscular dystrophy: Potential use as an endpoint in clinical trials. Gissy JJ; Johnson T; Fox DJ; Kumar A; Ciafaloni E; van Essen AJ; Peay HL; Martin A; Lucas A; Finkel RS; Neuromuscul Disord; 2017 Oct; 27(10):905-910. PubMed ID: 28739181 [TBL] [Abstract][Full Text] [Related]