Learn about the promising advance in prenatal treatment of spinal muscular atrophy, introducing a potential approach to suppress symptoms of the disorder in infants.

In its most severe form, as in the case of this child, individuals lack both copies of the SMN1 gene, and have only one or two copies of a neighbouring gene, SMN2, that partially compensates for ...

In general, each corresponds to the copy number of the SMN2 gene, which produces a small fraction (~10%) of functional SMN protein, essential to the survival of motor neurons, compared with SMN1.