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June 2021; 11 (3) ResearchOpen Access

Nusinersen Treatment in Adults With Spinal Muscular Atrophy

Tina Duong, Connie Wolford, Michael P. McDermott, Chelsea E. Macpherson, Amy Pasternak, Allan M. Glanzman, William B. Martens, Elizabeth Kichula, Basil T. Darras, Darryl C. De Vivo, Zarazuela Zolkipli-Cunningham, Richard S. Finkel, Michael Zeineh, Max Wintermark, Jacinda Sampson, Katharine A. Hagerman, Sally Dunaway Young, John W. Day
First published January 25, 2021, DOI: https://doi.org/10.1212/CPJ.0000000000001033
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Figures

  • Figure 1
    Figure 1 Clinical Outcomes Based on Functional Groups

    6MWT = 6-minute walk test; CHOP-ATEND = Children's Hospital of Philadelphia's Adult Test of Neuromuscular Disorders; FVC = forced vital capacity; HFMSE = Expanded Hammersmith Functional Motor Scale; MEP = maximal expiratory pressure; MIP = maximal inspiratory pressure; PROM = patient-reported outcome measure; RULM = revised upper limb module; SMAFRS = Spinal Muscular Atrophy Functional Rating Scale; TUG = timed up and go.

  • Figure 2
    Figure 2 Average Annual Rate of Change in CHOP-ATEND

    Children's Hospital of Philadelphia Adult Test for Neuromuscular Disorders (CHOP-ATEND) results for the entire population studied (n = 24) Individual subject trajectories are shown. The dark line (with standard error bars) depicts the average trajectory, as estimated from a linear mixed-effects model.

  • Figure 3
    Figure 3 HFMSE Total Score Trajectories Based on Functional Status

    Hammersmith Functional Motor Scale Expanded (HFMSE) results for entire population studied (n = 31) grouped by functional status at baseline: nonsitters (n = 9), sitters (n = 11), and walkers (n = 11).

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