Longitudinal motor function and biomarker correlates in treated adult spinal muscular atrophy: a single-center cohort study - Takeaways - MDSpire

Longitudinal motor function and biomarker correlates in treated adult spinal muscular atrophy: a single-center cohort study

  • By

  • Chikashi Yano

  • Masahiro Ando

  • Akiko Yoshimura

  • Yujiro Higuchi

  • Yoshikatsu Noda

  • Jun-Hui Yuan

  • Tomonori Nakamura

  • Takahiro Hobara

  • Risa Nagatomo

  • Fumikazu Kojima

  • Mika Yuji

  • Yu Hiramatsu

  • Satoshi Nozuma

  • Akihiro Hashiguchi

  • Yusuke Sakiyama

  • Hiroshi Takashima

  • June 11, 2026

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  • 1

    This study evaluated long-term motor function and biomarker associations in 23 adults with spinal muscular atrophy treated with nusinersen or risdiplam.

  • 2

    Higher SMN2 copy number correlated with later onset and milder disease severity in adults with spinal muscular atrophy.

  • 3

    Motor scores improved or stabilized during the first year of treatment, followed by a plateau or decline in some patients over a mean follow-up of 57 months.

  • 4

    Vital capacity, creatine kinase, and ulnar CMAP amplitude were identified as clinically accessible biomarkers associated with motor function.

  • 5

    Prediction of treatment responsiveness in spinal muscular atrophy remains challenging despite the identification of potential biomarkers.

Original Source(s)

Longitudinal motor function and biomarker correlates in treated adult spinal muscular atrophy: a single-center cohort study

  • by Chikashi Yano, Masahiro Ando, Akiko Yoshimura, Yujiro Higuchi, Yoshikatsu Noda, Jun-Hui Yuan, Tomonori Nakamura, Takahiro Hobara, Risa Nagatomo, Fumikazu Kojima, Mika Yuji, Yu Hiramatsu, Satoshi Nozuma, Akihiro Hashiguchi, Yusuke Sakiyama, Hiroshi Takashima

  • June 11, 2026

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