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Isis therapy for spinal muscular atrophy provides encouraging results

  • Isis Pharmaceuticals' (ISIS) ISIS-SMN treatment appears to have increased muscle function in an ongoing multiple-dose Phase 1b/2a study of children with spinal muscular atrophy, interim results demonstrate.
  • In addition, results from a biomarker assay show an increase of SMN protein (Survival of Motor Neuron) in the cerebral spinal fluid. The protein is particularly important for the maintenance of motor-neuron nerve cells, which control muscle movement.
  • The results give Isis "further confidence to advance ISIS-SMNRx into a Phase 3 program" says CFO Lynne Parshall. That trial is due to start later this year. (PR)
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