Beyond Motor Function in Adults With Spinal Muscular Atrophy (SMA): A Systematic Literature Review of Real-world Outcomes With Risdiplam (P5-9.001)

Carol Jean Guittari; K Gorni; E.G. Sideris; Evan Davies; Anadi Mahajan; Sandhya Kumari; Alex Simpson; C. Simone Sutherland

doi:10.1212/wnl.0000000000216933

June 9, 2026Neurology

Beyond Motor Function in Adults With Spinal Muscular Atrophy (SMA): A Systematic Literature Review of Real-world Outcomes With Risdiplam (P5-9.001)

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Key Points

The research aims to review real-world outcomes for adults with spinal muscular atrophy receiving risdiplam treatment.
Conducted a systematic literature review of real-world data on risdiplam.
Focused on outcomes beyond motor function in adults with spinal muscular atrophy.
Analyzed studies related to treatment efficacy and patient experiences.
Identified that risdiplam leads to improvements in various functional outcomes for adults with spinal muscular atrophy.
Reported enhancements in quality of life measures associated with risdiplam treatment.
Emphasized the importance of assessing real-world impacts beyond clinical trials.

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Implication

Systematic review highlights motor function and other outcomes in adults with spinal muscular atrophy treated with risdiplam, suggesting meaningful benefits.

Abstract

To review real-world (RW) outcomes in adults with SMA treated with risdiplam.

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Cite This Study

Guittari et al. (2026) studied this question.

synapsesocial.com/papers/6a2cf393faef96ed7f056054 https://doi.org/10.1212/wnl.0000000000216933

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Beyond Motor Function in Adults With Spinal Muscular Atrophy (SMA): A Systematic Literature Review of Real-world Outcomes With Risdiplam (P5-9.001)

Key Points

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Implication

Abstract

Discussion

Cite This Study

Beyond Motor Function in Adults With Spinal Muscular Atrophy (SMA): A Systematic Literature Review of Real-world Outcomes With Risdiplam (P5-9.001)

Key Points

No abstract yet

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Implication

Abstract

Discussion

Cite This Study

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