Nusinersen (Spinraza) for spinal muscular atrophy.
D006801:Humans; D009043:Motor Activity / Q000187:drug effects; D009134:Muscular Atrophy, Spinal / Q000188:drug therapy* / Q000235:genetics / Q000378:metabolism / Q000503:physiopathology; D009841:Oligonucleotides / Q000009:adverse effects / Q000627:therapeutic use*; D020127:Recovery of Function; D055540:Survival of Motor Neuron 2 Protein / Q000096:biosynthesis / Q000235:genetics; D016896:Treatment Outcome
D009841:Oligonucleotides; C526477:SMN2 protein, human; D055540:Survival of Motor Neuron 2 Protein; C000590926:nusinersen