From: N-terminal titin fragment: a non-invasive, pharmacodynamic biomarker for microdystrophin efficacy
Group | Microdystrophin expression cutoff based on %positive fibers by IF | n | Average age at dosing (years) +/- SD | p value | Average baseline titin (RFU) +/- SD | p value | Average baseline CK (U/L) +/- SD | p value | Average %positive fibers by IF post day 90 administration | p value |
---|---|---|---|---|---|---|---|---|---|---|
1 |  < 10% | 5 | 7.2 +/- 3.8 | 0.9590 | 36,106 +/- 41,196 | 0.8480 | 9131 +/- 4355 | 0.2556 | 2% +/- 4.47% | 0.0045 |
2 |  > 10% | 3 | 7.3 +/- 2.3 | 41,096 +/- / 10,172 | 12,876 +/- 3467 | 47% +/- 23.09% |