Severity of SMA Muscle Impairment Prior to Treatment May Have Implications for Nusinersen Efficacy

A new study has found that the severity of muscle impairment and scoliosis before treatment may help predict response to nusinersen for certain patients with spinal muscular atrophy (SMA).

The retrospective study of patients with SMA type 2 and 3, published in Brain and Development, found that the severity of fatty infiltration and muscle atrophy at the start of treatment had implications for changes in motor function after treatment with nusinersen. The findings, which the researchers say highlight the importance of early diagnosis and initiation of treatment, come amid efforts to determine efficacy trends among the several treatment options for the condition.