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Biochemical and clinical biomarkers in adult SMA 3–4 patients treated with nusinersen for 22 months

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To investigate biomarkers of disease progression in cerebrospinal fluid (CSF) and serum in adult patients with spinal muscular atrophy (SMA). Furthermore, we assess the clinical response to nusinersen treatment in… Click to show full abstract

To investigate biomarkers of disease progression in cerebrospinal fluid (CSF) and serum in adult patients with spinal muscular atrophy (SMA). Furthermore, we assess the clinical response to nusinersen treatment in adults with SMA over a longer follow‐up period than the previously reported 6–14 months.

Keywords: adult sma; biochemical clinical; clinical biomarkers; patients treated; sma patients; biomarkers adult

Journal Title: Annals of Clinical and Translational Neurology
Year Published: 2022

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