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