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A radically new treatment for SMA

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Until now there have been no treatments for spinal muscular atrophy (SMA) that have had any effect on the progressive muscle weakness. It has been known for years that the primary defect in the SMN1 gene exerts its effects through failure to produce adequate amounts of SMN protein in the motor neuron cells. However a nearly identical gene, SMN2, may remain intact, and could be induced to produce sufficient of the protein to restore some motor function. A new drug, nusinersen, is a uniformly modified antisense oligonucleotide which has been specifically developed to bind to repressive sites on the …

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  • Competing interests None declared.

  • Provenance and peer review Commissioned; internally peer reviewed.