SMA THERAPIES I

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We previously showed that treatment by nusinersen in spinal muscular atrophy (SMA) type 1 patients older than 7 months may have comparable effects to those observed in the younger population, but the response to the treatment is highly variable. In our cohort, median improvement in the modified motor milestones Hammersmith infant neurological exam (HINE-2) score was 1.5 points after 6 months of treatment and 2 points after 10 months. We still lack long-term clinical data and predictive factors for clinical benefit in this cohort.

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