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Gene Therapy Improves Muscle Function for Children With Spinal Muscular Atrophy
Children with spinal muscular atrophy (SMA) carry specific gene defects which cause progressive muscle wasting and weakness. Left untreated, the condition usually results in early death. Approval was recently granted for a gene replacement therapy that enables the maintenance of muscle function. However, due to the treatment’s relative novelty, data on safety and efficacy remain limited.
A team from Charité – Universitätsmedizin Berlin recently conducted the first multicenter clinical study to evaluate data on the side effects associated with this new gene replacement therapy. Their study showed that treatment resulted in significant improvement in muscle function, particularly in children under two years of age. The researchers’ findings have been published in The Lancet Child & Adolescent Health.
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