Biochemical and clinical biomarkers in adult SMA 3-4 patients treated with nusinersen for 22 months.
Bram De WelMaxim De SchaepdryverKoen PoesenKristl G ClaeysPublished in: Annals of clinical and translational neurology (2022)
YKL-40 in CSF correlated with clinical improvements during nusinersen treatment. In contrast, CHIT1 and pNfH in CSF changed significantly during treatment but did not correlate with clinical outcomes. Finally, we demonstrated a sustained clinical effect of nusinersen treatment in adults after 22 months.