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Sonia654

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Apr 11, 2021
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Spinraza is a medication used in treating spinal muscular atrophy (SMA), a rare neuromuscular disorder. Spinraza is a medicine used to treat 5q spinal muscular atrophy (SMA), a genetic disease that causes weakness and wasting of the muscles including the lung muscles. The disease is linked to a defect on chromosome 5q and symptoms usually start shortly after birth. Because the number of patients with SMA is low, the disease is considered ‘rare’, and Spinraza was designated an ‘orphan medicine’ (a medicine used in rare diseases).
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