Targeted Treatment for Type 1 Spinal Muscle Atrophy
Nusinersen, a drug used to treat spinal muscle atrophy in children as well as adults has shown remarkable effects in a study, the results of which were published in the New England Journal of Medicine. The study was conducted in 121 children affected with spinal muscle atrophy to evaluate the efficacy and safety of nusinersen. While some children received nusinersen, others received a placebo that served as a control.
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