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

Author(s):

Lauren Elman, MD,Arun S. Varadhachary, MD, PhD

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.

EP: 1.Clinical Features and Phenotypes of Spinal Muscular Atrophy

EP: 2.Natural History of Spinal Muscular Atrophy

EP: 3.Presentation of Adult Spinal Muscular Atrophy in Clinics

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

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

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

EP: 7.Treatments Approved for Treatment of Spinal Muscular Atrophy

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

EP: 9.Switching Treatments in Spinal Muscular Atrophy

EP: 10.Determining Treatment Success in Spinal Muscular Atrophy

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

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

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

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

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