First drug to treat spinal muscular atrophy gets FDA approval: Costly injectable shown to stall debilitating muscle-wasting effects of rare genetic disorder.
D002648:Child; D017277:Drug Approval / Q000191:economics; D006801:Humans; D009134:Muscular Atrophy, Spinal / Q000188:drug therapy* / Q000191:economics / Q000235:genetics; D009154:Mutation; D009841:Oligonucleotides / Q000191:economics / 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
D009841:Oligonucleotides; C526471:SMN1 protein, human; C526477:SMN2 protein, human; D055533:Survival of Motor Neuron 1 Protein; D055540:Survival of Motor Neuron 2 Protein; C000590926:nusinersen