Compound Muscle Action Potentials as Potential Motor Outcome Predictors of Spinal Muscular Atrophy in Children with Onasemnogene Abeparvovec-xioi Gene Therapy (S24.004)

Sneha Sadhu; Bahadir Samur

doi:10.1212/wnl.0000000000213089

June 9, 2026Neurology

Compound Muscle Action Potentials as Potential Motor Outcome Predictors of Spinal Muscular Atrophy in Children with Onasemnogene Abeparvovec-xioi Gene Therapy (S24.004)

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Key Points

The aim is to explore CMAP amplitudes as potential biomarkers for disease severity in neonates with spinal muscular atrophy.
Investigation of CMAP amplitudes in neonates with spinal muscular atrophy identified through newborn screening and confirmed by genetic testing.
Findings suggest that CMAP amplitudes correlate with disease severity in treated neonates.

Implication

Investigates CMAP amplitudes as disease severity biomarkers in neonates receiving gene therapy for SMA, suggesting clinical implications.

Abstract

This study aims to investigate the potential utility of compound muscle action potential (CMAP) amplitudes as biomarkers for assessing disease severity in neonates with spinal muscular atrophy (SMA) identified by newborn screening (NBS) and confirmed by genetic testing.

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Cite This Study

Sadhu et al. (2026) studied this question.

synapsesocial.com/papers/6a2cf32afaef96ed7f0559d3 https://doi.org/10.1212/wnl.0000000000213089

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Compound Muscle Action Potentials as Potential Motor Outcome Predictors of Spinal Muscular Atrophy in Children with Onasemnogene Abeparvovec-xioi Gene Therapy (S24.004)

Key Points

Implication

Abstract

Discussion

Cite This Study

Compound Muscle Action Potentials as Potential Motor Outcome Predictors of Spinal Muscular Atrophy in Children with Onasemnogene Abeparvovec-xioi Gene Therapy (S24.004)

Key Points

Implication

Abstract

Discussion

Cite This Study

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