● Improvements in motor function in patients from the SUNFISH study, observed in the first year of oral treatment, persisted into the fourth year.
● Both patients and their caregivers noted steady improvement or at least stabilization in their ability to perform daily activities, such as eating, drinking, combing their hair, brushing their teeth or fastening buttons independently.
● Adverse events and serious adverse events were related to the disease itself, not
to the treatment. The percentage of adverse events decreased over 48 months.
The SUNFISH clinical trial was one of the studies that registered the oral drug risdiplam for use in patients with spinal muscular atrophy (SMA). The study involved 180 patients between the ages of 2 and 25 years old with SMA type 2, i.e., those who never started walking independently, and with SMA type 3, who have lost the ability to walk independently....
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