Why the study?
Can close monitoring and adaptation of immunosuppressive regimens control the immune response in patients with Spinal Muscular Atrophy treated with onasemnogene abeparvovec?
Can close monitoring and adaptation of immunosuppressive regimens control the immune response in patients with Spinal Muscular Atrophy treated with onasemnogene abeparvovec?
Immune responses to onasemnogene abeparvovec in SMA patients can be managed with close monitoring and tailored immunosuppression.
Requires close monitoring and regimen adaptation for onasemnogene abeparvovec; hypothesis-generating and should not yet change practice.
In a broader spectrum of patients, treatment with onasemnogene abeparvovec was associated with a higher rate of adverse events. In our cases it was possible to control the immune response by close monitoring and adaptation of the immunosuppressive regimen. Further research is needed to better understand the immune response following gene therapy and ideally to identify patients at risk for a more severe reaction.
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Friese et al. (2021) studied this question.
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