title: FDA Approval of Nusinersen for Spinal Muscular Atrophy Makes 2016 the Year of Splice Modulating Oligonucleotides.
authors.affiliation: Department of Human Genetics, Leiden University Medical Center , Leiden, the Netherlands .
authors.forename: Annemieke
authors.initials: A
authors.lastname: Aartsma-Rus
mesh_terms: D000293:Adolescent; D000328:Adult; D017398:Alternative Splicing / Q000187:drug effects*; D000818:Animals; D002648:Child; D002675:Child, Preschool; D017277:Drug Approval; D006801:Humans; D007223:Infant; D023421:Models, Animal; D060172:Morpholinos / Q000627:therapeutic use; D009134:Muscular Atrophy, Spinal / Q000188:drug therapy* / Q000191:economics; D009841:Oligonucleotides / Q000191:economics / Q000494:pharmacology / Q000627:therapeutic use*; D016376:Oligonucleotides, Antisense / Q000191:economics / Q000494:pharmacology / Q000627:therapeutic use*; D055533:Survival of Motor Neuron 1 Protein / Q000235:genetics; D055540:Survival of Motor Neuron 2 Protein / Q000235:genetics; D014481:United States; D014486:United States Food and Drug Administration
chemical_list: D060172:Morpholinos; D009841:Oligonucleotides; D016376:Oligonucleotides, Antisense; C526471:SMN1 protein, human; C526477:SMN2 protein, human; D055533:Survival of Motor Neuron 1 Protein; D055540:Survival of Motor Neuron 2 Protein; C000590926:nusinersen; C000611335:eteplirsen
keywords: antisense oligonucleotide; eteplirsen; nusinersen; spinal muscular atrophy; therapy