SMA therapies (a LinkAGE webinar)

[@GenomicsEducation]

TIMESTAMPS 00:00 Introductions and talk overview 02:57 What is SMA? About its types and genomics 12:50 What symptoms should clinicians look out for for the different SMA types? 19:39 Intro of nusinersen, risdiplam and Zolgensma treatments 21:44 Clinical trials - an overview 23:25 Endear: a nusinersen clinical trial in babies (in depth) 30:42 The Fish trials for risdiplam (Rainbow-, Fire-, Sun-, Jewel-fish) 36:13 Zolgensma: overview, treatment, side effects, considerations 41:57 Treatment benefits of nusinersen, risdiplam and Zolgensma 42:11 About the presymptomatic clinical trials (Spr1nt, Rainbowfish, Nurture) 45:55 What are we seeing from real-world data? 50:31 New understandings: SMN gene and SMA disease progression 56:11 Combination therapies 57:21 Q&A and wrap up