Cerebrospinal Fluid and Clinical Profiles in Adult Type 2-3 Spinal Muscular Atrophy Patients Treated with Nusinersen: An 18-Month Single-Centre Experience.

Giammarco Milella Alessandro Introna Eustachio D'ErricoAngela FraddosioGaspare ScaglioneAntonella MoreaMaria UcciMaddalena RuggieriMariangela MastrapasquaMarisa Megna Filomena Puntillo Isabella Laura Simone

Published in: Clinical drug investigation (2021)

The increase of the Qalb values and the development of systemic OCBs in some SMA patients could be due to repeated lumbar puncture and to the immunogenic effect of nusinersen. On the other hand, the presence of OCBs in serum and/or CSF at baseline should be further investigated. Furthermore, biomarkers of neurodegeneration did not play a prognostic role in our cohort of adult SMA patients.

Keyphrases

end stage renal disease
newly diagnosed
ejection fraction
chronic kidney disease
cerebrospinal fluid
prognostic factors
peritoneal dialysis