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June 9, 2026Neurology

Real-world Motor Function Outcomes with Risdiplam in Types 2 and 3 Spinal Muscular Atrophy (SMA): A Systematic Literature Review and Meta-analysis (P5-9.002)

Carol Jean Guittari Evan Davies C. Simone Sutherland K Gorni Susan Edwards Maya Kjærgaard Anadi Mahajan Digant Gupta Mudasir Khan Sandyha Kumari E.G. Sideris

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Key Points

This review aims to assess motor function outcomes in individuals with Types 2 and 3 spinal muscular atrophy treated with risdiplam.
Systematic literature review and meta-analysis of real-world outcomes.
Focus on motor function improvements in SMA Types 2 and 3.
Evaluation of data from multiple studies on risdiplam treatment.
Motor function showed improvement in individuals treated with risdiplam.
Significant changes in motor function measures were noted.
Positive trends were consistent across reviewed studies.

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Implication

Systematic review reveals motor function improvements in Types 2 and 3 spinal muscular atrophy after risdiplam treatment.

Abstract

To review real-world (RW) motor function outcomes in individuals with Types 2 and 3 SMA treated with risdiplam.

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Cite This Study

Guittari et al. (2026) studied this question.

synapsesocial.com/papers/6a2cf393faef96ed7f05605f https://doi.org/10.1212/wnl.0000000000216891

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