Extracellular vesicles as biomarkers of disease progression and therapeutic response in patients with spinal muscular atrophy.

As shown in Figure 3 A, similar to our findings in plasma, we observed a trend toward increased NP concentration in CSF from individuals with SMA compared to healthy controls, although this difference did not reach statistical significance.

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Normalization of CSF NP concentration data to the value obtained at baseline for all patients allowed us to analyze the average relative concentration of NPs in the CSF, which showed a trend toward a very modest decrease in NP concentration over the course of dosing treatment, which did not achieve statistical significance ( Figure 3 F).

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