title: Antisense rescues babies from killer disease.
authors.forename: Meredith
authors.initials: M
authors.lastname: Wadman
mesh_terms: D035843:Biomedical Research / Q000639:trends; D001927:Brain Diseases / Q000235:genetics* / Q000628:therapy*; D002675:Child, Preschool; D017322:Clinical Trials, Phase II as Topic; D006801:Humans; D009134:Muscular Atrophy, Spinal / Q000235:genetics* / Q000628:therapy*; D009841:Oligonucleotides / Q000627:therapeutic use*; D016372:RNA, Antisense / Q000627:therapeutic use*; D055533:Survival of Motor Neuron 1 Protein / Q000235:genetics; D055540:Survival of Motor Neuron 2 Protein / Q000235:genetics
chemical_list: D009841:Oligonucleotides; D016372:RNA, 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