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Treating SMA: moving beyond genetic approaches

As a result of dedicated efforts by the pharma industry, there are now two drugs approved for SMA: Spinraza and Zolgensma. Although these have been life-changing for patients with this debilitating condition, they are not perfect. More treatments are needed for this condition; Cure SMA notes the importance of developing therapies that focus beyond the SMN genes underlying the condition, and towards the processes affected by the degeneration.


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