To evaluate the efficacy and safety of efgartigimod in the treatment of ALS in China.

A total of 58 patients had their ALSFRS-R scores recorded both at baseline and after treatment, including 19 with bulbar-onset ALS and 39 with spinal-onset ALS. The baseline ALSFRS-R scores ranged from 15 to 45. A majority (70.6%) of the patients received a minimum of two injections of efgartigimod. The mean follow-up period was 8 weeks. Improvements in ALSFRS-R scores were observed in 46.5% (27/58) of the patients, while 24.1% (14/58) exhibited stable scores. The overall response rate was 70.6%, with comparable rates between bulbar-onset and spinal-onset ALS, although the improvement rate was notably higher in spinal-onset ALS. Long-term treatment with efgartigimod appears to slow the progression of ALS without additional adverse events. Further detailed data will be presented at the conference.

In this study, we observed the certain efficacy and safety of efgartigimod in treatment of ALS. The study is still in progress, with ongoing collection of efficacy and safety data from a larger cohort. We intend to further investigate the relationship between patients' antibody test results and the outcomes.