Emerging Therapeutic Strategies for Spinal Muscular Atrophy: A Comprehensive Review

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Zolgensma resulted in improved event-free survival and maintained respiratory function, marking a breakthrough in SMA therapy.
Risdiplam approximately doubled SMN protein levels, leading to notable improvements in motor function across age groups.
Nusinersen reduced risk of mortality and permanent ventilation, showcasing its potential impact on survival for SMA patients.
Overall, these treatments demonstrate significant benefits, but further research on long-term safety remains essential.

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Comprehensive review highlights gene therapies and their significant effects on motor function in spinal muscular atrophy patients.

Nagaraju et al. (2024) studied this question.

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