1. Updates in SMA From an International Muscle Meeting

3. This program will include a discussion of data that were presented in abstract form. These data should be considered preliminary until published in a peer-reviewed journal.

4. Spinal Muscular Atrophy Overview

5. Types of Infant-Onset SMA

6. Clinical and Economic Burden of SMA

7. Demographic Profile and Clinical Characteristics Cure SMA Database

8. Survival Estimates for Type I and Type II SMA

9. Economic Burden of Type I SMA Retrospective Claims Analysis

10. Potential Biomarkers for SMA

11. pNF-H Level as a Biomarker of Disease Activity

12. pNF-H Levels in SMA

13. Biomarkers for SMA Clinical Implications

14. Optimizing Outcomes With Current Strategies for SMA

15. Survival and Ventilation Use in Type I SMA Analysis of Data From Cure SMA Survey, 2017

16. Survival and Ventilation Use in Type I SMA Survey Results

17. Nusinersen

18. SHINE Study Interim Analysis Patients Transitioned From ENDEAR

19. SHINE Study Endpoints

20. SHINE Study Interim Analysis Head Control and Independent Sitting

21. SHINE Study Interim Analysis Key Learnings

22. Emerging Clinical Data

23. AVXS-101

24. AVXS-101: Phase 1 Study

25. AVXS-101 Results at 24-Month Follow-Up Age at Dosing and Motor Milestone Achievement

26. Risdiplam

27. Risdiplam SMN2 Splicing Modifier

28. SUNFISH Part 1 Results After 12 Months of Treatment

29. JEWELFISH Results After 4 Weeks of Treatment

30. FIREFISH Part 1 Improvement in CHOP-INTEND Scores

31. FIREFISH Part 1 Achievement of Motor Skills After 8 Months

32. Branaplam

33. Branaplam First-in-Human Dose-Escalation Study

34. Abbreviations