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

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.

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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