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Review
. 2024 Aug 7;32(8):2489-2504.
doi: 10.1016/j.ymthe.2024.06.020. Epub 2024 Jun 18.

Clinical perspectives: Treating spinal muscular atrophy

Molly A McPheron  1 , Marcia V Felker  2
Affiliations
Review

Clinical perspectives: Treating spinal muscular atrophy

Molly A McPheron et al. Mol Ther. .

Abstract

Spinal muscular atrophy is a rare and progressive neuromuscular disease that, without treatment, leads to progressive weakness and often death. A plethora of studies have led to the approval of three high-cost and effective treatments since 2016. These treatments, nusinersen, onasemnogene abeparvovec, and risdiplam, have not been directly compared and have varying challenges in administration. In this review, we discuss the evidence supporting the use of these medications, the process of treatment selection, monitoring after treatment, the limited data comparing treatments, as well as future directions for investigation and therapy.

Keywords: gene replacement therapy; neuromuscular; nusinersen; onasemnogene abeparvovec; risdiplam; spinal muscular atrophy.

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Conflict of interest statement

Declaration of interests The authors declare no competing interests.

Figures

None
Graphical abstract
Figure 1
Figure 1
SMN1 and SMN2 genes
See this image and copyright information in PMC

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