Fatal outcomes following onasemnogene abeparvovec in advanced-stage spinal muscular atrophy

This single-center, real-world observational study examined the outcomes of five SMA type 1 patients and three SMA type 2 patients who received OA gene replacement therapy over a three-year period. Our investigation predominantly addressed advanced-stage SMA type 1 patients whose treatment was delayed due to drug authorization and transportation issues. Unfortunately, all patients in this advanced group died, highlighting the critical need to reevaluate the timing and appropriateness of gene therapy in such patients.

Symptom onset in our SMA…