Table 3 Efficacy rate for investigator's integrated efficacy assessment (FAS)
From: Efficacy confirmation study of aceneuramic acid administration for GNE myopathy in Japan
Treatment Group | N | n | Effective | Ineffective | Undeterminable | Efficacy Rate (%) | 95% CI for Rate | ||
|---|---|---|---|---|---|---|---|---|---|
Lower | Upper | ||||||||
SA-ER | 10 | 10 | 7 | 3 | 0 | 70.0 | 34.75 | – | 93.33 |
Placebo | 4 | 4 | 2 | 2 | 0 | 50.0 | 6.76 | – | 93.24 |