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Volumn 24, Issue 9, 2017, Pages 534-538

New treatments for serious conditions: Ethical implications

Author keywords

[No Author keywords available]

Indexed keywords

NUSINERSEN; OLIGONUCLEOTIDE;

EID: 85032483155     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2017.32     Document Type: Review
Times cited : (33)

References (25)
  • 1
  • 2
    • 34548190615 scopus 로고    scopus 로고
    • Spinal muscular atrophy: Clinical classification and disease heterogeneity
    • Russman BS. Spinal muscular atrophy: clinical classification and disease heterogeneity. J Child Neurol 2007; 22: 946-951.
    • (2007) J Child Neurol , vol.22 , pp. 946-951
    • Russman, B.S.1
  • 3
    • 0028067907 scopus 로고
    • The natural history of type 1 (severe) spinal muscular atrophy
    • Thomas NH, Dubowitz V. The natural history of type 1 (severe) spinal muscular atrophy. Neuromuscul Disord 1994; 4: 497-502.
    • (1994) Neuromuscul Disord , vol.4 , pp. 497-502
    • Thomas, N.H.1    Dubowitz, V.2
  • 4
    • 84945953905 scopus 로고    scopus 로고
    • Spinal muscular atrophy
    • Kolb SJ, Kissle JT. Spinal muscular atrophy. Neurol Clin 2015; 33: 831-846.
    • (2015) Neurol Clin , vol.33 , pp. 831-846
    • Kolb, S.J.1    Kissle, J.T.2
  • 5
    • 84960517144 scopus 로고    scopus 로고
    • Results from a phase 1 study of nusinersen (ISIS-SMN Rx) in children with spinal muscular atrophy
    • Chiriboga CA, Swoboda KJ, Darras BT, Iannaccone ST, Hontes J, De Vivo DC et al. Results from a phase 1 study of nusinersen (ISIS-SMN Rx) in children with spinal muscular atrophy. Neurology 2016; 86: 890-897.
    • (2016) Neurology , vol.86 , pp. 890-897
    • Chiriboga, C.A.1    Swoboda, K.J.2    Darras, B.T.3    Iannaccone, S.T.4    Hontes, J.5    De Vivo, D.C.6
  • 6
    • 85006321439 scopus 로고    scopus 로고
    • Treatment of infantile-onset spinal muscular atrophy with nusinersen: A phase 2, open-label, dose-escalation study
    • Finkel RS, Chiriboga CA, Vajsar J, Day JW, Montes J, De Vivo DC et al. Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study. Lancet 2016; 388: 3017-3026.
    • (2016) Lancet , vol.388 , pp. 3017-3026
    • Finkel, R.S.1    Chiriboga, C.A.2    Vajsar, J.3    Day, J.W.4    Montes, J.5    De Vivo, D.C.6
  • 7
    • 85032511510 scopus 로고    scopus 로고
    • Biogen Press Release, 2016. US (accessed on 4 February
    • Biogen Press Release, 2016. US. FDA approves Biogen's Spinraza (nusinersen), the first treatment for spinal muscular atrophy. Available at: http://media.biogen.com/ press-release/neurodegenerative-diseases/us-fda-approves-biogens-spinrazanusinersen-first-treatment (accessed on 4 February 2017).
    • (2017) FDA Approves Biogen's Spinraza (Nusinersen), the First Treatment for Spinal Muscular Atrophy
  • 8
    • 85019679806 scopus 로고    scopus 로고
    • FDA News Release, 2016 (accessed on 4 February
    • FDA News Release, 2016. FDA approves first drug for spinal muscular atrophy. Available at: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ ucm534611.htm (accessed on 4 February 2017).
    • (2017) FDA Approves First Drug for Spinal Muscular Atrophy
  • 9
    • 84969287518 scopus 로고    scopus 로고
    • Intrathecal injections in children with spinal muscular atrophy: Nusinersen clinical trial experience
    • Hache M, Swoboda KJ, Sethna N, Farrow-Gillespie A, Khandji A, Xia S et al. Intrathecal injections in children with spinal muscular atrophy: nusinersen clinical trial experience. J Child Neurol 2016; 3: 899-906.
    • (2016) J Child Neurol , vol.3 , pp. 899-906
    • Hache, M.1    Swoboda, K.J.2    Sethna, N.3    Farrow-Gillespie, A.4    Khandji, A.5    Xia, S.6
  • 11
    • 24944522335 scopus 로고    scopus 로고
    • Confronting the issues of therapeutic misconception, enrollment decisions, and personal motives in genetic medicine-based clinical research studies for fatal disorders
    • Arkin LM, Sondhi D, Worgall S, Suh LHK, Hackett NR, Kaminsky SM et al. Confronting the issues of therapeutic misconception, enrollment decisions, and personal motives in genetic medicine-based clinical research studies for fatal disorders. Hum Gene Ther 2005; 16: 1028-1036.
    • (2005) Hum Gene Ther , vol.16 , pp. 1028-1036
    • Arkin, L.M.1    Sondhi, D.2    Worgall, S.3    Suh, L.H.K.4    Hackett, N.R.5    Kaminsky, S.M.6
  • 15
    • 85032491297 scopus 로고    scopus 로고
    • Investopedia, 4 January 2017. (accessed on 5 February
    • Shobit S. Biogen, Ionis under fire for Spinraza. Investopedia, 4 January 2017. Available at: http://www.investopedia.com/news/biogen-ionis-under-fire-spinraza-biib-ions/?ad = dirN&qo = investopediaSiteSearch&qsrc = 0&o = 40186 (accessed on 5 February 2017).
    • (2017) Biogen, Ionis under Fire for Spinraza
    • Shobit, S.1
  • 17
    • 85032511175 scopus 로고    scopus 로고
    • Biogen, SMA 360. (accessed on 5 February
    • Biogen, SMA 360. Available at: https://www.spinraza.com/en-us/home/patientsupport-services/biogen-support-program.html (accessed on 5 February 2017).
    • (2017)
  • 18
    • 85032500841 scopus 로고    scopus 로고
    • Genetic Information and Nondiscrimination Act, 2008. Pub L No 110-233
    • Genetic Information and Nondiscrimination Act, 2008. Pub L No 110-233
  • 20
    • 0343354500 scopus 로고
    • The technology of medicine
    • Viking Press: New York, NY, USA
    • Thomas L. The technology of medicine. In: Lives of a Cell: Notes of a Biology Watcher. Viking Press: New York, NY, USA, 1974, pp 31-36.
    • (1974) Lives of A Cell: Notes of A Biology Watcher , pp. 31-36
    • Thomas, L.1
  • 21
    • 84928091454 scopus 로고    scopus 로고
    • A mixed methods exploration of families' experiences of the diagnosis of childhood spinal muscular atrophy
    • Lawton S, Hickerton C, Archibald AD, McClaren BJ, Metcalfe SA. A mixed methods exploration of families' experiences of the diagnosis of childhood spinal muscular atrophy. Eur J Hum Genet 2015; 23: 575-580.
    • (2015) Eur J Hum Genet , vol.23 , pp. 575-580
    • Lawton, S.1    Hickerton, C.2    Archibald, A.D.3    McClaren, B.J.4    Metcalfe, S.A.5
  • 22
    • 85032507704 scopus 로고    scopus 로고
    • The want of control: Ideas and ideals in the management of diabetes
    • UNC Press: Chapel Hill, NC, USA
    • Feudtner C. The want of control: ideas and ideals in the management of diabetes. In: Bittersweet: Diabetes, Insulin, and the Transformation of Illness. UNC Press: Chapel Hill, NC, USA, 2003, pp 121-145.
    • (2003) Bittersweet: Diabetes, Insulin, and the Transformation of Illness , pp. 121-145
    • Feudtner, C.1
  • 23
    • 84939125157 scopus 로고    scopus 로고
    • Ethical considerations in tissue engineering research: Case studies in translation
    • Baker HB, McQuilling JP, King NMP. Ethical considerations in tissue engineering research: case studies in translation. Methods 2016; 99: 135-144.
    • (2016) Methods , vol.99 , pp. 135-144
    • Baker, H.B.1    McQuilling, J.P.2    King, N.M.P.3
  • 25
    • 84923817475 scopus 로고    scopus 로고
    • Enduring and emerging challenges of informed consent
    • Grady C. Enduring and emerging challenges of informed consent. N Engl J Med 2015; 372: 855-862.
    • (2015) N Engl J Med , vol.372 , pp. 855-862
    • Grady, C.1


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.