MTT | Exondys 51 |
1. 100% dystrophin production | 0.28% dystrophin m-RNA increase |
2. 70% muscle fiber increase and histology improved | None |
3. 123% strength increase in 18 months | None |
4. 48.7% decrease in serum CPK in 1 year | None |
5. 19% increase in breathing capacity | None |
6. Physical capacity improved | None |
7. Life prolonged | None |
8. For DMD, BMD and other MDs | For 13% DMD with Exon 51 skipping |