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Opinion|Videos|July 16, 2024

Real-World Data and Stability with Nusinersen in Adult Patients with Spinal Muscular Atrophy

Author(s)Lauren Elman, MD, Arun S. Varadhachary, MD, PhD , Thomas Crawford, MD

Key opinion leaders address the scarcity of randomized controlled trial data for SMA treatments in adult populations, highlighting the availability of real-world data from numerous studies and provide guidance on how to incorporate this information when making treatment decisions for adult patients with spinal muscular atrophy.

Episodes in this series

Video 1 - 5 KOLs are featured in "Clinical Features and Phenotypes of Spinal Muscular Atrophy"

EP. 1 Clinical Features and Phenotypes of Spinal Muscular Atrophy

Video 2 - 5 KOLs are featured in "Natural History of Spinal Muscular Atrophy"

EP. 2 Natural History of Spinal Muscular Atrophy

Video 3 - 5 KOLs are featured in "Presentation of Adult Spinal Muscular Atrophy in Clinics"

EP. 3 Presentation of Adult Spinal Muscular Atrophy in Clinics

Video 3 - 5 KOLs are featured in "Transitions of Care: Moving Spinal Muscular Atrophy Patients from Pediatric/Adolescent Care to Adult Clinics"

EP. 4 Transitions of Care: Moving Spinal Muscular Atrophy Patients from Pediatric/Adolescent Care to Adult Clinics

Video 5 - 5 KOLs are featured in "Key Players in Transitioning Patients with SMA from Pediatric Care to Adult Health Care"

EP. 5 Key Players in Transitioning Patients with SMA from Pediatric Care to Adult Health Care

Video 5 - 5 KOLs are featured in "Managing Clinical Stability in Adults Spinal Muscular Atrophy Patients"

EP. 6 Managing Clinical Stability in Adults Spinal Muscular Atrophy Patients

5 KOLs are featured in this series.

EP. 7 Treatments Approved for Treatment of Spinal Muscular Atrophy

5 KOLs are featured in this series.

EP. 8 Factors Guiding Treatment Selection in Adult Spinal Muscular Atrophy

5 KOLs are featured in this series.

EP. 9 Switching Treatments in Spinal Muscular Atrophy

5 KOLs are featured in this series.

EP. 10 Determining Treatment Success in Spinal Muscular Atrophy

5 KOLs are featured in this series.

EP. 11 Counseling Patients About Treatment Outcomes in Spinal Muscular Atrophy

5 KOLs are featured in this series.

EP. 12 Real-World Data and Stability with Nusinersen in Adult Patients with Spinal Muscular Atrophy

5 KOLs are featured in this series.

EP. 13 Real-World Data for Risdiplam in Adult Patients with Spinal Muscular Atrophy

5 KOLs are featured in this series.

EP. 14 Transition of Care in Young Adults With Spinal Muscular Atrophy

5 KOLs are featured in this series.

EP. 15 Recommendations for Clinicians Taking Care of Patients with SMA

While we do not have randomized controlled trial data from adults for these agents, real world-data (RWD) is available from multiple studies with nusinersen (and a few with risdiplam) in adults worldwide.
1. Please share your basic understanding of the RWE available for nusinersen real-world studies

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