Beyond motor rescue: defining adequate SMN restoration in spinal muscular atrophy.

Cell-specific SMN expression and persistent peripheral immune cell alterations are demonstrated in nusinersen- or risdiplam-treated adults with spinal muscular atrophy, reframing what “adequate” SMN restoration means.

• Why it matters: Motor neuron rescue alone is no longer sufficient to explain residual disease biology after therapy, and the field needs clearer criteria for adequate SMN restoration across cell types and time.
• What they did: Tapken, Rahmel-Stein, and colleagues assessed cell-specific SMN expression and profiled peripheral immune cell populations in adults with SMA receiving nusinersen or risdiplam.
• The result: The work suggests selective vulnerability extends beyond motor neurons and supports a therapeutic framework that considers cell type, developmental timing, SMN requirements, and downstream responses for systemic and CNS-directed approaches.

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