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Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy

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Nusinersen (Spinraza) is a recently approved drug for treating spinal muscular atrophy. Approval of nusinersen may signal new opportunities for using antisense oligonucleotides as treatments for devastating neurological diseases. Click to show full abstract

Nusinersen (Spinraza) is a recently approved drug for treating spinal muscular atrophy. Approval of nusinersen may signal new opportunities for using antisense oligonucleotides as treatments for devastating neurological diseases.

Keywords: spinal muscular; drug; antisense oligonucleotide; nusinersen antisense; muscular atrophy

Journal Title: Nature Neuroscience
Year Published: 2017

Link to full text (if available)


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