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Roche’s aim to widen Evrysdi’s label nabs speedy FDA review in young spinal muscular atrophy patients

Now poised for a speedy trip through the FDA's regulatory review, Roche’s oral challenger in spinal muscular atrophy (SMA) could soon pick up a nod to cover all patie...
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Scotland Leads the Way in Approval of First Licensed Treatment for Life-Threatening Genetic Disease Affecting Children

Routine access to Spinraza® (nusinersen) for type 1 spinal muscular atrophy granted, with discussions continuing to secure access for other forms of the disease The S...
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