Managing Spinal Muscular Atrophy in Adult Patients : Episode 12
Real-World Data and Stability with Nusinersen in Adult Patients with Spinal Muscular Atrophy
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.
- Please share your basic understanding of the RWE available for nusinersen real-world studies
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