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JAMA Neurology
Volumn 73, Issue 3, 2016, Pages 259-260
Antisense oligonucleotides for duchenne muscular dystrophy
Author keywords

[No Author keywords available]
Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; CORTICOSTEROID; DRISAPERSEN; DYSTROPHIN; ETEPLIRSEN;
EID: 84961695483     PISSN: 21686149     EISSN: None     Source Type: Journal    
DOI: 10.1001/jamaneurol.2015.4011     Document Type: Note
Times cited : (4)
References (7)
  • 1
    • 84992408411 scopus 로고    scopus 로고
    • Silver Spring, MD: Food and Drug Administration Center for Drug Evaluation and Research US Department of Health and Human Services Food and Drug Administration
    • US Department of Health and Human Services Food and Drug Administration. Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment. Silver Spring, MD: Food and Drug Administration Center for Drug Evaluation and Research; 2015.
    • (2015) Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment
  • 2
    • 0031800293 scopus 로고    scopus 로고
    • Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides
    • Dunckley MG, Manoharan M, Villiet P, Eperon IC, Dickson G. Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides. Hum Mol Genet. 1998;7(7):1083-1090.
    • (1998) Hum Mol Genet , vol.7 , Issue.7 , pp. 1083-1090
    • Dunckley, M.G.1    Manoharan, M.2    Villiet, P.3    Eperon, I.C.4    Dickson, G.5
  • 3
    • 0035878539 scopus 로고    scopus 로고
    • Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells
    • van Deutekom JC, Bremmer-Bout M, Janson AA, et al. Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells. Hum Mol Genet. 2001;10(15):1547-1554.
    • (2001) Hum Mol Genet , vol.10 , Issue.15 , pp. 1547-1554
    • Van Deutekom, J.C.1    Bremmer-Bout, M.2    Janson, A.A.3
  • 4
    • 84890805770 scopus 로고    scopus 로고
    • Eteplirsen for the treatment of Duchenne muscular dystrophy
    • Eteplirsen Study Group
    • Mendell JR, Rodino-Klapac LR, Sahenk Z, et al; Eteplirsen Study Group. Eteplirsen for the treatment of Duchenne muscular dystrophy. Ann Neurol. 2013;74(5):637-647.
    • (2013) Ann Neurol , vol.74 , Issue.5 , pp. 637-647
    • Mendell, J.R.1    Rodino-Klapac, L.R.2    Sahenk, Z.3
  • 5
    • 79955158683 scopus 로고    scopus 로고
    • Systemic administration of PRO051 in Duchenne's muscular dystrophy
    • Goemans NM, Tulinius M, van den Akker JT, et al. Systemic administration of PRO051 in Duchenne's muscular dystrophy. N Engl J Med. 2011;364(16): 1513-1522.
    • (2011) N Engl J Med , vol.364 , Issue.16 , pp. 1513-1522
    • Goemans, N.M.1    Tulinius, M.2    Van Den Akker, J.T.3
  • 6
    • 84907991204 scopus 로고    scopus 로고
    • Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): An exploratory, randomised, placebo-controlled phase 2 study
    • Voit T, Topaloglu H, Straub V, et al. Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): An exploratory, randomised, placebo-controlled phase 2 study. Lancet Neurol. 2014;13(10):987-996.
    • (2014) Lancet Neurol , vol.13 , Issue.10 , pp. 987-996
    • Voit, T.1    Topaloglu, H.2    Straub, V.3
  • 7
    • 61649097962 scopus 로고    scopus 로고
    • Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations
    • Aartsma-Rus A, Fokkema I, Verschuuren J, et al. Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations. Hum Mutat. 2009;30(3):293-299.
    • (2009) Hum Mutat , vol.30 , Issue.3 , pp. 293-299
    • Aartsma-Rus, A.1    Fokkema, I.2    Verschuuren, J.3

* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.