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Article

Nusinersen as a Therapeutic Agent for Spinal Muscular Atrophy

1 Jan 2020

Abstract excerpt

The reduction of survival motor neuron (SMN) protein causes spinal muscular atrophy (SMA), an autosomal recessive neuromuscular disease. Nusinersen is an antisense oligonucleotide, approved by the FDA, which specifically binds to the repressor within SMN2 exon 7 to enhance exon 7 inclusion and augment production of functional SMN protein. Nusinersen is the first new oligonucleotide-based drug targeting the...

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PMID: 32233169
DOI: 10.3349/ymj.2020.61.4.273

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