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September 24, 2019; 93 (13) ArticleOpen Access

Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function

Eric P. Hoffman, Benjamin D. Schwartz, Laurel J. Mengle-Gaw, Edward C. Smith, Diana Castro, Jean K. Mah, Craig M. McDonald, Nancy L. Kuntz, Richard S. Finkel, Michela Guglieri, Katharine Bushby, Mar Tulinius, Yoram Nevo, Monique M. Ryan, Richard Webster, Andrea L. Smith, Lauren P. Morgenroth, Adrienne Arrieta, Maya Shimony, Catherine Siener, Mark Jaros, Phil Shale, John M. McCall, Kanneboyina Nagaraju, John van den Anker, Laurie S. Conklin, Avital Cnaan, Heather Gordish-Dressman, Jesse M. Damsker, Paula R. Clemens, the Cooperative International Neuromuscular Research Group
First published August 26, 2019, DOI: https://doi.org/10.1212/WNL.0000000000008168
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Article Figures & Data

Figures

  • Figure 1
    Figure 1 Primary endpoint time to stand mean (±SE) velocity change from baseline

    Brackets indicate mixed-model repeated-measures p values (black for comparisons to Cooperative International Neuromuscular Research Group Duchenne Natural History Study [DNHS] external comparator; blue for within-trial dose group comparisons).

  • Figure 2
    Figure 2 Time to run/walk 10 m mean (±SE) velocity change from baseline

    Brackets indicate mixed-model repeated-measures p values (black for comparisons to Cooperative International Neuromuscular Research Group Duchenne Natural History Study [DNHS] external comparator; blue for within-trial dose group comparisons).

  • Figure 3
    Figure 3 Six-minute walk test mean (±SE) change from baseline

    Brackets indicate mixed-model repeated-measures p values (within-trial dose group comparisons).

  • Figure 4
    Figure 4 Time to climb mean (±SE) velocity change from baseline

    Brackets indicate mixed-model repeated-measures p values (black for comparisons to Cooperative International Neuromuscular Research Group Duchenne Natural History Study [DNHS] external comparator).

  • Figure 5
    Figure 5 Body mass index (BMI) z score mean (±SE) change from baseline (safety population)

    Brackets indicate mixed-model repeated-measures p values (comparisons to Cooperative International Neuromuscular Research Group [CINRG] prednisone trial external comparator).

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