An overview of the history of SMA is provided, highlighting its evolving classification and the genetic basis underlying its clinical variability, and the development and clinical trial data of the three Food and Drug Administration- and European Medicines Agency-approved therapies: nusinersen, onasemnogene abeparvovec, and risdiplam are outlined.
Olcay Ünver, J. Wilmshurst, Haluk A. Topaloglu· Journal of International Chi...· 0 citations
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