A multinational clinical trial involving 26 infants in seven countries examined the effects of risdiplam, an SMN2 pre-mRNA splicing modifier, on infants genetically confirmed with spinal muscular atrophy (SMA) but without clinical symptoms. The infants, aged 1 to 42 days at dosing, showed significant motor milestone achievements after 24 months of treatment, with 21 able to sit unsupported for 30 seconds and 11 able to walk independently. The study suggested that early risdiplam treatment supports survival and motor function in SMA, though larger studies are necessary for confirmation.
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