Key result
Serial CMAP amplitudes assess improvement following onasemnogene abeparvovec-xioi therapy in early-onset SMA.
Why the study?
This study was conducted to evaluate the utility of serial compound muscle action potential amplitudes in early-onset spinal muscular atrophy before and after onasemnogene abeparvovec-xioi gene replacement therapy.
Does serial compound muscle action potential (CMAP) have utility in assessing improvement following onasemnogene abeparvovec-xioi gene replacement therapy in early-onset spinal muscular atrophy (SMA)?
Does serial compound muscle action potential (CMAP) have utility in assessing improvement following onasemnogene abeparvovec-xioi gene replacement therapy in early-onset spinal muscular atrophy (SMA)?
Serial CMAP monitoring may aid SMA gene therapy assessment; leaves open validation before clinical adoption.
This study aims to investigate the utility of serial compound muscle action potential (CMAP) amplitudes in early-onset spinal muscular atrophy (SMA) before and after onasemnogene abeparvovec-xioi gene replacement therapy.
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Samur et al. (2026) studied this question. Serial compound muscle action potential (CMAP) amplitudes were investigated to assess improvement before and after onasemnogene abeparvovec-xioi therapy in early-onset SMA.
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