1. Best Practices in SMA

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

3. Overview of SMA

4. Clinical Characteristics of SMA

5. Validated Outcome Measures in SMA

6. Genetic Basis of SMA

7. SMN2 Copy Number Is a Primary Determinant of Disease Severity

8. Current and Investigational Targeted Therapies for SMA

9. Nusinersen

10. ENDEAR: Study Design

11. ENDEAR: Endpoints

12. ENDEAR: Event-Free and Overall Survival

13. ENDEAR: HINE-2 Responses

14. ENDEAR: CHOP-INTEND Results

15. ENDEAR: Safety

16. Nusinersen in SMA: Early-Phase Studies

17. CHERISH: Study Design

18. CHERISH: Results

19. CHERISH: Results (cont)

20. NURTURE: Study Design and Results

21. HINE Motor Milestone Scores Across Studies

22. Newborn Screening for SMA in the United States

23. Early Diagnosis and Interventions in SMA

24. SMN-Targeting Agents in the Pipeline

25. AVXS-101: Gene Replacement Therapy

26. AVXS-101: Motor Function Results

27. AVXS-101: Motor Response in High-Dose Group

28. Risdiplam: SMN2 Splicing Modifier

29. Reldesemtiv: Troponin Activator

30. SRK-015: Myostatin Inhibitor

31. Moving New Research Into the Clinic

32. Looking Ahead: Open Research Issues

33. Abbreviations