Clinical Outcome of Adult Spinal Muscular Atrophy Patients Treated with Nusinersen: A Case Series Review.

INTRODUCTION: Nusinersen is an antisense oligonucleotide drug that was developed for treatment of spinal muscular atrophy (SMA). Its effectiveness for adults is limited; therefore, more clinical data are needed to guide adult SMA patients who are considering this treatment. METHODS: Through case series review, we retrospectively reviewed charts of Kaiser Permanente Southern California members who were already receiving nusinersen treatment, which has been available since August 2017. Patients were evaluated by physical therapy using the Hammersmith Functional Motor Scale (out of highest possible score of 40). RESULTS: We identified 4 adult patients who met our study criteria as of February 1, 2020. All patients were mobility device dependent. Patient age ranged from 23 to 56 years. A generalized linear model was used to assess trendlines for repeated measures within subjects. In this small sample, there appears to a significant increase in scores on repeated measures (p = 0.0027). CONCLUSION: Based on this small study, some adult SMA patients may benefit from treatment with nusinersen.