Abstract
Recently, the anti-sense oligonucleotide drug nusinersen was approved for spinal muscular atrophy (SMA). Our aim was to evaluate the development in motor function of the patients receiving treatment and to investigate response markers in CSF for this treatment. All patients with SMA started on treatment with nusinersen during the period of November 2016 – June 2020 at Queen Silvia Children's Hospital were included in the study. Clinical data were obtained from the patients’ medical records. Motor function was assessed by standardized motor function scales validated for patients with SMA.
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