British clinicians report their real-life experience of two children who received onasemnogene replacement gene therapy abeparvovec (OA) at a later age than usual:
- these two children with type 1 spinal muscular atrophy had received nusinersen for several years and were aged 7 and weighed 20 kg at the time of the OA injection,
- in addition to the fact that the functional efficacy of OA was not achieved, or was only transient,
- numerous side-effects, mainly in the liver, necessitated long-term corticosteroid therapy and, in the case of one of the children, long-term immunosuppression with tacrolimus.
The authors warn against raising expectations too high when this type of additional therapy is administered at an advanced stage of the disease and/or at a high weight.
