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Risdiplam, one of three therapies approved for spinal muscular atrophy (SMA) and the only oral option, is a pre-mRNA splicing modulator that increases functional survival of motor neuron 2 (SMN2) protein. The aim of the current analysis of an ongoing, international, open-label, industry-funded trial was to assess the 24-month safety and efficacy of daily risdiplam for type 1 SMA, following a report of 12-month outcomes (N Engl J Med 2021; 385:427). The dose was adjusted for age. The median age at enrollment was 5.3 months (interquartile range, 4.2–6.8 months); disease duration was >3 months in 66%. Out of 41 participants, 3 died due to SMA-related respiratory complications before 12 months and 38 completed 24 months of treatment. Exclusions…