FDA Approval of Nusinersen for Spinal Muscular Atrophy Makes 2016 the Year of Splice Modulating Oligonucleotides

Abstract excerpt

With the Food and Drug Administration (FDA) approval of nusinersen (tradename Spinraza) for treatment of spinal muscular atrophy patients on December 23 [1], 2016 brought us not one, but two approved splice modulating oligonucleotides to treat neuromuscular disorders.Eteplirsen, an oligonucleotide to treat a subset of Duchenne muscular dystrophy (DMD) patients, was approved earlier this year.However, where the...

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