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Muscle MRI as an Endpoint in Clinical Trials

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Muscle Gene Therapy

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Abstract

In the last decade, there has been substantial progress in the genetic characterization and classification of inherited muscle disorders. In addition to clinical assessment, clinical neurophysiology, and the diagnostic gold standard of histopathology including immunohistochemistry, muscle imaging, and particularly magnetic resonance imaging (MRI), has increasingly been used in the diagnostic work-up of inherited muscle disease. Novel quantitative muscle MRI techniques have been developed in order to characterize and quantify the severity and pattern of muscle involvement in clinical routine as well as in therapeutic trials. This chapter provides a comprehensive overview of current MRI techniques in inherited muscle diseases with special emphasis on the use of quantitative muscle MRI in clinical therapeutic trials.

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Author information

Authors and Affiliations

  1. Division of Neuropaediatrics, University Children’s Hospital Basel, University of Basel, Basel, Switzerland

    Dirk Fischer & Ulrike Bonati

  2. Department of Neurology, University Hospital Basel, University of Basel, Basel, Switzerland

    Dirk Fischer & Ulrike Bonati

  3. Department of Diagnostic and Interventional Neuroradiology, Hannover Medical School, Hannover, Germany

    Mike P. Wattjes

Authors
  1. Dirk Fischer
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  2. Ulrike Bonati
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  3. Mike P. Wattjes
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Corresponding author

Correspondence to Dirk Fischer .

Editor information

Editors and Affiliations

  1. Department of Molecular Microbiology and Immunology, School of Medicine, University of Missouri, Columbia, MO, USA; Department of Neurology, School of Medicine, University of Missouri, Columbia, MO, USA, Department of Biomedical Sciences College of Veterinary Medicine, University of Missouri, Columbia, MO, USA; Department of Bioengineering, University of Missouri, Columbia, MO, USA

    Dongsheng Duan

  2. Department of Pediatrics Nationwide Children’s Hospital and Research Institute, Department of Neurology Nationwide Children’s Hospital and Research Institute, The Ohio State University, Columbus, OH, USA

    Jerry R. Mendell

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Fischer, D., Bonati, U., Wattjes, M.P. (2019). Muscle MRI as an Endpoint in Clinical Trials. In: Duan, D., Mendell, J. (eds) Muscle Gene Therapy. Springer, Cham. https://doi.org/10.1007/978-3-030-03095-7_40

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