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Spinal muscular atrophy (SMA) is an autosomal recessive disease characterized by progressive muscle weakness due to the loss of anterior horn motor neurons. Nearly all cases of SMA are caused by homozygous deletion or mutation of the SMN1 gene. SMA ranges widely in severity, and symptom onset usually occurs during childhood. In its most serious form, SMA is lethal in infancy. Although no treatments yet exist for SMA, several exciting therapies in late-phase development may enter the market in the near term, and several others are in earlier stages of clinical development. The bulk of the pipeline focuses on correcting the underlying defect in SMA—i.e., the deficient expression of the SMN protein.