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Breakthrough Gene Therapy Boosts Motor Function in SMA Kids

3/19/2025

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Summary

Story summary

At the 2025 MDA Conference, the phase 3 STEER study revealed that intrathecal onasemnogene abeparvovec significantly enhances motor function in children aged 2-17 with spinal muscular atrophy. The study showcased remarkable gains in the Hammersmith Functional Motor Scale, while also highlighting a favorable safety profile, paving the way for broader gene therapy access for older patients.

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Story summary

At the 2025 MDA Conference, the phase 3 STEER study revealed that intrathecal onasemnogene abeparvovec significantly enhances motor function in children aged 2-17 with spinal muscular atrophy. The study showcased remarkable gains in the Hammersmith Functional Motor Scale, while also highlighting a favorable safety profile, paving the way for broader gene therapy access for older patients.