Wendy Chung, Kennedy Family Professor of Medicine in Pediatrics at Columbia University
Chapters:
0:00 Studies that transformed treatment of SMA led to the GUARDIAN study
5:45 Overall goals
8:35 Latest results
15:35 Is the study making the case for whole genome sequencing at birth?
21:55 Challenges in becoming standard of care
31:35 Concerns?
37:20 What puts a s…