The FDA has approved a tablet formulation of Evrysdi® (risdiplam), a survival motor neuron 2 pre-mRNA splicing modifier, for the treatment of spinal muscular atrophy (SMA). The tablet provides comparable systemic exposure, efficacy, and safety as the oral solution, and offers the advantage of room temperature stability, eliminating the need for refrigeration. This new option provides patients aged 2 years and older with an alternative administration method for the treatment of SMA.
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