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May 21, 2019; 92 (21) ArticleOpen Access

Nusinersen in later-onset spinal muscular atrophy

Long-term results from the phase 1/2 studies

Basil T. Darras, Claudia A. Chiriboga, Susan T. Iannaccone, Kathryn J. Swoboda, Jacqueline Montes, Laurence Mignon, Shuting Xia, C. Frank Bennett, Kathie M. Bishop, Jeremy M. Shefner, Allison M. Green, Peng Sun, Ishir Bhan, Sarah Gheuens, Eugene Schneider, Wildon Farwell, Darryl C. De Vivo
First published April 24, 2019, DOI: https://doi.org/10.1212/WNL.0000000000007527
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Article Figures & Data

Figures

  • Figure 1
    Figure 1 CS2 (phase 1b/2a open-label) and CS12 (extension) study designs and patient disposition

    (A) Study designs: aOverall enrollment. bPatients received treatment on days 1 and 85 only. (B) Patient disposition: The CS2-CS12 integrated efficacy analysis included 28 children from CS2 who received their first dose of nusinersen in CS2 and could have been treated in CS12. The total number of nusinersen doses administered in CS2 was 2 (cohort 3) or 3 (cohorts 1, 2, and 4), and the total number of nusinersen doses administered in CS12 was 4. D = day; SMA = spinal muscular atrophy.

  • Figure 2
    Figure 2 Mean change from baseline in Hammersmith Functional Motor Scale–Expanded (HFMSE) score

    Due to the gap between CS2 and CS12 and windowing of visit days, some windowed visits do not contain all children. SE = standard error; SMA = spinal muscular atrophy.

  • Figure 3
    Figure 3 Mean change from baseline in Upper Limb Module (ULM) score and 6-Minute Walk Test (6MWT) distance

    (A) ULM: Assessed only in nonambulant participants. (B) 6MWT: Assessed only in ambulant participants. Due to the gap between CS2 and CS12 and windowing of visit days, some windowed visits do not contain all children. SE = standard error.

  • Figure 4
    Figure 4 Mean change from baseline in compound muscle action potential (CMAP) amplitude, CMAP area, and motor unit number estimation (MUNE) value

    (A) CMAP amplitude, (B) CMAP area, and (C) MUNE value. Due to the gap between CS2 and CS12 and windowing of visit days, some windowed visits do not contain all children. SE = standard error; SMA = spinal muscular atrophy.

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