1. Importance of Early Diagnosis and Management in SMA

2. Spinal Muscular Atrophy

3. Most Common SMA 5q Type

4. Family History

5. What Does SMA Look Like? Clinical Signs of Disease

6. Historic Diagnosis Correlates With Genetics

7. Understanding the Genetics of SMA

8. Understanding the Genetics of SMA (cont)

9. Historic Diagnosis Correlates With SMN2 Copy Number

10. Diagnostic Testing for SMA Has Improved Significantly With Genetic Advancements

11. Natural History of Disease Patient With Type 1 SMA

12. Respiratory Weakness

13. Standards of Care Statements

14. Heterogeneous and Complex Disorder

15. Managing Respiratory Complications

16. Managing Orthopedic Complications

17. Managing Nutritional Health

18. SMA Therapies

19. Gene Transfer Therapy vs SMN2 Modification

20. Characteristics of Onasemnogene Abeparvovec-xioi

21. Clinical Trial in SMA Type 1 Onasemnogene Abeparvovec-xioi

22. Clinical Trial in SMA Type 1 Onasemnogene Abeparvovec-xioi (cont)

23. Clinical Trial in SMA Type 1 Nusinersen

24. Newborn Screening

25. Newborn Screening Nationwide Status as of February 2019

26. Conclusions

27. Abbreviations

28. Abbreviations (cont)