Why the study?
What are the current and emerging targeted therapies for spinal muscular atrophy?
What are the current and emerging targeted therapies for spinal muscular atrophy?
Targeted therapies for SMA, including gene therapies and splice modulators, have transformed the disease management from palliative care to disease-modifying treatments that improve survival and motor function.
Approved SMA therapies benefit symptomatic and pre-symptomatic patients; leaves open need for durable single-dose options.
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Lakhina et al. (2023) studied this question.
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