1. New Horizons for SMA

2. This program will include a discussion of investigational agents not approved by the FDA for use in the US, and data that were presented in abstract form. These data should be considered preliminary until published in a peer-reviewed journal.

3. Introduction to Spinal Muscular Atrophy

4. Introduction to Spinal Muscular Atrophy (cont)

5. Why Treat SMA?

6. Burden of SMA Type 1

7. Burden of SMA Types 2 and 3

8. Clinical Trial Endpoints

9. Assessing Outcomes in Clinical Practice

10. Mechanisms of Action of New Therapies

11. SMA Treatment Mechanism of Action

12. SMA Treatment Mechanism of Action (cont)

13. Nusinersen

14. ENDEAR Trial Design

15. ENDEAR Trial Results

16. ENDEAR: Motor Milestone Responders

17. ENDEAR: CHOP INTEND Motor Function

18. Change in HINE Score From Baseline

19. Change in CHOP-INTEND Score From Baseline

20. CHERISH Trial Design

21. CHERISH Trial: HFMSE Scores

22. Phase 1 Trial of AVXS-101

23. Phase 1 Trial of AVXS-101 (cont)

24. Efficacy in Phase 1 Trial of AVXS-101

25. Olesoxime: Mechanism of Action

26. Olesoxime: Efficacy and Safety

27. Maintaining Motor Function in SMA

28. Agents That May Improve Nerve or Muscle Function

29. Conclusions

30. Abbreviations