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Summary Combining bioinformatic and drug repurposing approaches to develop second-generation therapies for spinal muscular atrophy Spinal muscular atrophy (SMA) is a devastating childhood disease caused by loss of function of the survival motor neuron (SMN) gene, leading to the death of nerve and muscle cells. The first SMN gene replacement therapies have recently been approved […]

  • Education
  • Community and government

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