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Interventions for muscular dystrophy: molecular medicines entering the clinic.

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  • Additional Information
    • Affiliation:
      Institute of Human Genetics, Newcastle University, Newcastle upon Tyne, UK
      Institute of Human Genetics, Newcastle University, Newcastle upon Tyne, UK.
    • Subject Terms:
    • Subject Terms:
    • Abstract:
      Muscular dystrophies are individually rare genetic disorders that cause much chronic disability, affecting young children and adults. In the past 20 years, more than 30 genetic types of muscular dystrophy have been defined. During this time, precise diagnosis, genetic counselling, and medical management have improved. These advances in medical practice have occurred while definitive therapies based on an improved knowledge of disease pathogenesis are awaited. A wide range of therapeutic options have been tested in animal models, and some are being tested in clinical trials. Various therapeutic targets are being investigated, from personalised medicines targeting specific mutations and drugs targeting cellular pathways to gene-based and cell-based therapies.
    • Journal Subset:
      Biomedical; Editorial Board Reviewed; Europe; Expert Peer Reviewed; Peer Reviewed; UK & Ireland
    • ISSN:
      0099-5355
    • MEDLINE Info:
      PMID: NLM19944865 NLM UID: 2985213R
    • Grant Information:
      G0601943//Medical Research Council/United Kingdom
    • Publication Date:
      20100115
    • Publication Date:
      20200708
    • DOI:
      10.1016/S0140-6736(09)61834-1
    • Accession Number:
      105255422
  • Citations
    • ABNT:
      BUSHBY K et al. Interventions for muscular dystrophy: molecular medicines entering the clinic. Lancet, [s. l.], v. 374 North American Edition, n. 3704, p. 1849–1856, 2009. DOI 10.1016/S0140-6736(09)61834-1. Disponível em: http://search.ebscohost.com/login.aspx?direct=true&site=eds-live&db=rzh&AN=105255422. Acesso em: 25 out. 2020.
    • AMA:
      Bushby K, Lochmüller H, Lynn S, et al. Interventions for muscular dystrophy: molecular medicines entering the clinic. Lancet. 2009;374 North American Edition(3704):1849-1856. doi:10.1016/S0140-6736(09)61834-1
    • APA:
      Bushby K, Lochmüller H, Lynn S, Straub V, Bushby, K., Lochmüller, H., Lynn, S., & Straub, V. (2009). Interventions for muscular dystrophy: molecular medicines entering the clinic. Lancet, 374 North American Edition(3704), 1849–1856. https://doi.org/10.1016/S0140-6736(09)61834-1
    • Chicago/Turabian: Author-Date:
      Bushby K, Lochmüller H, Lynn S, Straub V, Kate Bushby, Hanns Lochmüller, Stephen Lynn, and Volker Straub. 2009. “Interventions for Muscular Dystrophy: Molecular Medicines Entering the Clinic.” Lancet 374 North American Edition (3704): 1849–56. doi:10.1016/S0140-6736(09)61834-1.
    • Harvard:
      Bushby K et al. (2009) ‘Interventions for muscular dystrophy: molecular medicines entering the clinic’, Lancet, 374 North American Edition(3704), pp. 1849–1856. doi: 10.1016/S0140-6736(09)61834-1.
    • Harvard: Australian:
      Bushby K, Lochmüller H, Lynn S, Straub V, Bushby, K, Lochmüller, H, Lynn, S & Straub, V 2009, ‘Interventions for muscular dystrophy: molecular medicines entering the clinic’, Lancet, vol. 374 North American Edition, no. 3704, pp. 1849–1856, viewed 25 October 2020, .
    • MLA:
      Bushby K, et al. “Interventions for Muscular Dystrophy: Molecular Medicines Entering the Clinic.” Lancet, vol. 374 North American Edition, no. 3704, Nov. 2009, pp. 1849–1856. EBSCOhost, doi:10.1016/S0140-6736(09)61834-1.
    • Chicago/Turabian: Humanities:
      Bushby K, Lochmüller H, Lynn S, Straub V, Kate Bushby, Hanns Lochmüller, Stephen Lynn, and Volker Straub. “Interventions for Muscular Dystrophy: Molecular Medicines Entering the Clinic.” Lancet 374 North American Edition, no. 3704 (November 28, 2009): 1849–56. doi:10.1016/S0140-6736(09)61834-1.
    • Vancouver/ICMJE:
      Bushby K, Lochmüller H, Lynn S, Straub V, Bushby K, Lochmüller H, et al. Interventions for muscular dystrophy: molecular medicines entering the clinic. Lancet [Internet]. 2009 Nov 28 [cited 2020 Oct 25];374 North American Edition(3704):1849–56. Available from: http://search.ebscohost.com/login.aspx?direct=true&site=eds-live&db=rzh&AN=105255422