Paradoxical increase of neurofilaments in SMA patients treated with onasemnogene abeparvovec-xioi

ConclusionSerum NfL showed a paradoxical transient increase after GRT in both, pre-treated and naïve patients, which may reflect an immunological reaction in the CNS related to transfection of neuronal cells by AAV9. The clinical meaning of this increase should be assessed in future studies. Our findings encourage regular monitoring of NfL in OA treated patients.
Source: Frontiers in Neurology - Category: Neurology Source Type: research