Panelists discuss how the current spinal muscular atrophy (SMA) treatment landscape includes 3 options: gene therapy (onasemnogene abeparvovec [Zolgensma]) for younger patients and 2 splice modifiers (nusinersen [Spinraza] and risdiplam [Evrysdi]) for older patients.
Clinical Brief: Historical and Current Treatment Paradigms for SMA
Main Discussion Topics
Key Points for Physicians
Notable Insights
Implementation of consensus care guidelines and multidisciplinary care improved survival outcomes even before disease-modifying therapies were available, highlighting the importance of comprehensive care approaches.
Clinical Significance
The treatment paradigm for SMA has evolved from palliative care to a multifaceted approach combining disease-modifying therapies with supportive care, requiring ongoing adaptation of assessment methods to capture meaningful clinical outcomes.
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