Abstract
Two manuscripts published recently in The Lancet Child and Adolescent Health, both address the efficacy of an advanced therapeutic strategy for hereditary neuromuscular diseases. Weiß et al. present real-world evidence of gene replacement therapy in spinal muscular atrophy (SMA).1 SMA is a hereditary lower motor neuron disease sharing a global incidence of around 1 in 10,000 live births.2 SMA was first described clinically in the 1890s but the first disease-modifying treatment, the antisense agent nusinersen (Spinraza), was only approved by the FDA in 2016.
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