Panelists discuss how splice modifiers work by enhancing protein production from the SMN2 gene, with risdiplam (Evrysdi) being an oral daily medication and nusinersen (Spinraza) being administered intrathecally quarterly, both showing similar safety and efficacy profiles.
Clinical Brief: Disease-Modifying Therapies for Spinal Muscular Atrophy (SMA)
Main Discussion Topics
Key Points for Physicians
Notable Insights
Patient preferences regarding treatment administration (daily oral medication vs quarterly intrathecal injection) may influence therapy selection, with some older patients preferring the less frequent dosing schedule of nusinersen despite its more invasive administration.
Clinical Significance
Selection of disease-modifying therapy for older patients with SMA currently involves choosing between nusinersen and risdiplam based on administration considerations, access to medical facilities, and patient preference, while gene therapy research continues for older populations.
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