Vaccination proposal for patients on onasemnogene abeparvovec therapy.

The approval of disease-modifying treatment in spinal muscular atrophy made the condition less severe. The course of the disease changed, but some new concerns occurred with the different new therapies. The side effects of onasemnogene aboparvovec therapy can raise differential diagnostic challenges and necessitate immune therapy, leading to immunosuppression affecting response to vaccines. We provide a pretherapy screening proposal from an infectological point of view separately for newborns treated presymptomatically and children diagnosed with symptoms at any age. Furthermore, we summarise the guidelines on the vaccination before, during, and after immune therapy (steroids) in onasemnogene aboparvovec-treated patients.

Informations de copyright

© 2024 Published by Elsevier Ltd on behalf of European Paediatric Neurology Society.

Déclaration de conflit d'intérêts

Declaration of competing interest All authors have disclosed all financial support for our work and other potential conflicts of interest.