Nusinersen, onasemnogene abeparvovec, and risdiplam are the only agents that have been approved for the treatment of SMA to date, but only nusinersen and risdiplam are approved for use in adult patients. Nusinersen is approved for all types of SMA, whereas risdiplam — which is taken orally — is approved for SMA types 1, 2 and 3.
Clinical data suggest nusinersen can be safely administered to adults with SMA with clinically meaningful benefits. In a recent retrospective review, the clinical benefits achieved with nusinersen in adults appeared to be cumulative over time. However, a high degree of variability in therapeutic response has also been observed, highlighting the need for biomarkers to better stratify patients to SMA treatments. Risdiplam has also shown encouraging results in adults with SMA.
Learn more about the treatment of SMA.
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Cite this: Stephen L. Nelson. Fast Five Quiz: Spinal Muscular Atrophy in Adults - Medscape - Feb 01, 2021.