메뉴 건너뛰기




Volumn 9, Issue 1, 2014, Pages

6 minute walk test in Duchenne MD patients with different mutations: 12 month changes

(44)  Pane, Marika a   Mazzone, Elena S a   Sormani, Maria Pia b   Messina, Sonia c   Vita, Gian Luca c   Fanelli, Lavinia a   Berardinelli, Angela d   Torrente, Yvan e   D'Amico, Adele f   Lanzillotta, Valentina g   Viggiano, Emanuela h   D'Ambrosio, Paola h   Cavallaro, Filippo c   Frosini, Silvia i   Bello, Luca j   Bonfiglio, Serena k   Scalise, Roberta a   De Sanctis, Roberto a   Rolle, Enrica l   Bianco, Flaviana a   more..


Author keywords

[No Author keywords available]

Indexed keywords

6 MINUTE WALK TEST; ADOLESCENT; ARTICLE; CHILD; COHORT ANALYSIS; DUCHENNE MUSCULAR DYSTROPHY; EXON SKIPPING; FUNCTION TEST; GENE DELETION; GENE DUPLICATION; GENE MUTATION; HUMAN; MAJOR CLINICAL STUDY; MALE; OBSERVATIONAL STUDY; OUTCOME ASSESSMENT; POINT MUTATION; WALKING;

EID: 84897449030     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0083400     Document Type: Article
Times cited : (75)

References (26)
  • 1
    • 82055196671 scopus 로고    scopus 로고
    • Molecular diagnosis of duchenne muscular dystrophy: Past, present and future in relation to implementing therapies
    • Laing NG, Davis MR, Bayley K, Fletcher S, Wilton SD (2011) Molecular diagnosis of duchenne muscular dystrophy: past, present and future in relation to implementing therapies. Clin Biochem Rev 32: 129-134.
    • (2011) Clin Biochem Rev , vol.32 , pp. 129-134
    • Laing, N.G.1    Davis, M.R.2    Bayley, K.3    Fletcher, S.4    Wilton, S.D.5
  • 2
    • 78650397994 scopus 로고    scopus 로고
    • New insights in gene-derived therapy: The example of Duchenne muscular dystrophy
    • Aartsma-Rus A, den Dunnen JT, van Ommen GJ (2010) New insights in gene-derived therapy: the example of Duchenne muscular dystrophy. Ann N Y Acad Sci 1214: 199-212.
    • (2010) Ann N Y Acad Sci , vol.1214 , pp. 199-212
    • Aartsma-Rus, A.1    Den Dunnen, J.T.2    Van Ommen, G.J.3
  • 3
    • 79960981599 scopus 로고    scopus 로고
    • Targeting RNA to treat neuromuscular disease
    • Muntoni F, Wood MJ (2011) Targeting RNA to treat neuromuscular disease. Nat Rev Drug Discov 10: 621-637.
    • (2011) Nat Rev Drug Discov , vol.10 , pp. 621-637
    • Muntoni, F.1    Wood, M.J.2
  • 4
    • 84864343619 scopus 로고    scopus 로고
    • Genetic therapeutic approaches for Duchenne muscular dystrophy
    • Foster H, Popplewell L, Dickson G (2012) Genetic therapeutic approaches for Duchenne muscular dystrophy. Hum Gene Ther 23: 676-687.
    • (2012) Hum Gene Ther , vol.23 , pp. 676-687
    • Foster, H.1    Popplewell, L.2    Dickson, G.3
  • 7
    • 84859867996 scopus 로고    scopus 로고
    • Overview on DMD exon skipping
    • Aartsma-Rus A (2012) Overview on DMD exon skipping. Methods Mol Biol 867: 97-116.
    • (2012) Methods Mol Biol , vol.867 , pp. 97-116
    • Aartsma-Rus, A.1
  • 8
    • 84862625633 scopus 로고    scopus 로고
    • Antisense oligonucleotide-mediated exon skipping for duchenne muscular dystrophy: Progress and challenges
    • Arechavala-Gomeza V, Anthony K, Morgan J, Muntoni F (2012) Antisense oligonucleotide-mediated exon skipping for duchenne muscular dystrophy: progress and challenges. Curr Gene Ther 12: 152-160.
    • (2012) Curr Gene Ther , vol.12 , pp. 152-160
    • Arechavala-Gomeza, V.1    Anthony, K.2    Morgan, J.3    Muntoni, F.4
  • 9
    • 77952009340 scopus 로고    scopus 로고
    • The development of antisense oligonucleotide therapies for Duchenne muscular dystrophy: Report on a TREAT-NMD workshop hosted by the European Medicines Agency (EMA), on September 25th 2009
    • Muntoni F (2010) The development of antisense oligonucleotide therapies for Duchenne muscular dystrophy: report on a TREAT-NMD workshop hosted by the European Medicines Agency (EMA), on September 25th 2009. Neuromuscul Disord 20: 355-362.
    • (2010) Neuromuscul Disord , vol.20 , pp. 355-362
    • Muntoni, F.1
  • 10
    • 80051690306 scopus 로고    scopus 로고
    • Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: An open-label, phase 2, dose-escalation study
    • Cirak S, Arechavala-Gomeza V, Guglieri M, Feng L, Torelli S, et al. (2011) Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study. Lancet 378: 595-605.
    • (2011) Lancet , vol.378 , pp. 595-605
    • Cirak, S.1    Arechavala-Gomeza, V.2    Guglieri, M.3    Feng, L.4    Torelli, S.5
  • 11
    • 84856546632 scopus 로고    scopus 로고
    • Restoration of the dystrophin-associated glycoprotein complex after exon skipping therapy in Duchenne muscular dystrophy
    • Cirak S, Feng L, Anthony K, Arechavala-Gomeza V, Torelli S, et al. (2012) Restoration of the dystrophin-associated glycoprotein complex after exon skipping therapy in Duchenne muscular dystrophy. Mol Ther 20: 462-467.
    • (2012) Mol Ther , vol.20 , pp. 462-467
    • Cirak, S.1    Feng, L.2    Anthony, K.3    Arechavala-Gomeza, V.4    Torelli, S.5
  • 13
    • 0141594934 scopus 로고    scopus 로고
    • Advances in Duchenne muscular dystrophy gene therapy
    • van Deutekom JC, van Ommen GJ (2003) Advances in Duchenne muscular dystrophy gene therapy. Nat Rev Genet 4: 774-783.
    • (2003) Nat Rev Genet , vol.4 , pp. 774-783
    • Van Deutekom, J.C.1    Van Ommen, G.J.2
  • 14
    • 77950210043 scopus 로고    scopus 로고
    • The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy
    • McDonald CM, Henricson EK, Han JJ, Abresch RT, Nicorici A, et al. (2010) The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy. Muscle Nerve 41: 500-510.
    • (2010) Muscle Nerve , vol.41 , pp. 500-510
    • McDonald, C.M.1    Henricson, E.K.2    Han, J.J.3    Abresch, R.T.4    Nicorici, A.5
  • 15
    • 80051478879 scopus 로고    scopus 로고
    • Functional changes in Duchenne muscular dystrophy: A 12-month longitudinal cohort study
    • Mazzone E, Vasco G, Sormani MP, Torrente Y, Berardinelli A, et al. (2011) Functional changes in Duchenne muscular dystrophy: a 12-month longitudinal cohort study. Neurology 77: 250-256.
    • (2011) Neurology , vol.77 , pp. 250-256
    • Mazzone, E.1    Vasco, G.2    Sormani, M.P.3    Torrente, Y.4    Berardinelli, A.5
  • 16
    • 84872223337 scopus 로고    scopus 로고
    • 24 month longitudinal data in ambulant boys with duchenne muscular dystrophy
    • Mazzone ES, Pane M, Sormani MP, Scalise R, Berardinelli A, et al. (2013) 24 month longitudinal data in ambulant boys with duchenne muscular dystrophy. PLoS One 8: e52512.
    • (2013) PLoS One , vol.8
    • Mazzone, E.S.1    Pane, M.2    Sormani, M.P.3    Scalise, R.4    Berardinelli, A.5
  • 17
    • 0036644465 scopus 로고    scopus 로고
    • ATS statement: Guidelines for the six-minute walk test
    • (2002) ATS statement: guidelines for the six-minute walk test. Am J Respir Crit Care Med 166: 111-117.
    • (2002) Am J Respir Crit Care Med , vol.166 , pp. 111-117
  • 18
    • 84860908668 scopus 로고    scopus 로고
    • Percent-predicted 6-minute walk distance in duchenne muscular dystrophy to account for maturational influences
    • Henricson E, Abresch R, Han JJ, Nicorici A, Goude Keller E, et al. (2012) Percent-predicted 6-minute walk distance in duchenne muscular dystrophy to account for maturational influences. PLoS Curr 4: RRN1297.
    • (2012) PLoS Curr , vol.4
    • Henricson, E.1    Abresch, R.2    Han, J.J.3    Nicorici, A.4    Goude Keller, E.5
  • 20
    • 80054720841 scopus 로고    scopus 로고
    • Genotype and phenotype characterization in a large dystrophinopathic cohort with extended follow-up
    • Magri F, Govoni A, D'Angelo MG, Del Bo R, Ghezzi S, et al. (2011) Genotype and phenotype characterization in a large dystrophinopathic cohort with extended follow-up. J Neurol 258: 1610-1623.
    • (2011) J Neurol , vol.258 , pp. 1610-1623
    • Magri, F.1    Govoni, A.2    D'Angelo, M.G.3    Del Bo, R.4    Ghezzi, S.5
  • 23
    • 84878746211 scopus 로고    scopus 로고
    • The incidence of revertant and trace dystrophin expression in muscle biopsies of Duchenne Muscular Dystrophy patients with different exon deletions
    • Lourbakos A, Sipkens J, Beekman C, Kreuger D, Brasz L, et al. (2011) The incidence of revertant and trace dystrophin expression in muscle biopsies of Duchenne Muscular Dystrophy patients with different exon deletions. Neuromuscul Disord 21: 643-643.
    • (2011) Neuromuscul Disord , vol.21 , pp. 643-643
    • Lourbakos, A.1    Sipkens, J.2    Beekman, C.3    Kreuger, D.4    Brasz, L.5
  • 24
    • 78751634526 scopus 로고    scopus 로고
    • SPP1 genotype is a determinant of disease severity in Duchenne muscular dystrophy
    • Pegoraro E, Hoffman EP, Piva L, Gavassini BF, Cagnin S, et al. (2011) SPP1 genotype is a determinant of disease severity in Duchenne muscular dystrophy. Neurology 76: 219-226.
    • (2011) Neurology , vol.76 , pp. 219-226
    • Pegoraro, E.1    Hoffman, E.P.2    Piva, L.3    Gavassini, B.F.4    Cagnin, S.5
  • 25
    • 84878390222 scopus 로고    scopus 로고
    • LTBP4 genotype predicts age of ambulatory loss in duchenne muscular dystrophy
    • e pub only
    • Flanigan KM, Ceco E, Lamar KM, Kaminoh Y, Dunn DM, et al. (2012) LTBP4 genotype predicts age of ambulatory loss in duchenne muscular dystrophy. Ann Neurol.2013 e pub only
    • (2012) Ann Neurol
    • Flanigan, K.M.1    Ceco, E.2    Lamar, K.M.3    Kaminoh, Y.4    Dunn, D.M.5
  • 26
    • 84883050768 scopus 로고    scopus 로고
    • The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: Reliability, concurrent validity, and minimal clinically important differences from a multicenter study
    • McDonald CM, Henricson EK, Abresch RT, Florence J, Eagle M, et al. (2013) The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: Reliability, concurrent validity, and minimal clinically important differences from a multicenter study. Muscle Nerve 48: 343-356.
    • (2013) Muscle Nerve , vol.48 , pp. 343-356
    • McDonald, C.M.1    Henricson, E.K.2    Abresch, R.T.3    Florence, J.4    Eagle, M.5


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.