Gene therapy medication provides improvements in severe SMA
Spinraza, the gene therapy medication, also provides significant improvements in cases with the next most severe form of the spinal muscular atrophy...
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Spinraza, the gene therapy medication, also provides significant improvements in cases with the next most severe form of the spinal muscular atrophy...
Infants with SMA are more likely to show gains in motor function and survive without assisted support when treated with nusinersen...