Patient Condition
The patient developed lower limb weakness since the age of 46, and was diagnosed with ALS at the age of 47. Genetic testing revealed a SOD1I113T mutation. At the time of diagnosis, ALSFRS-R was 34, FVC was 76%, and both lower limb muscle strength was grade 3+. The progress rate is moderate (0.9 points per month).
Treatment Timeline
| Phase | Details | Date |
|---|
| Tofersen treatment initiation | | |
| ongoing treatment for | | |
| long-term maintenance | | |
Treatment Outcome
| before | ALSFRS-R 34, monthly decline 0.9, FVC 76%, SOD1I113T mutation |
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| after | ALSFRS-R 30 points, 2-year decrease of 4 points (delay of 89%), FVC 70%, walking independently |
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| highlight | Tofersen targeted the SOD1I113T mutation to delay progression by 89%, with excellent results |
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