WRI Hospital Plays Key Role in Drug Approval

Cystic Fibrosis patients with the defect G551D mutation may benefit from the FDA’s approval of Kalydeco. Kalydeco is the first drug that targets the underlying causes of Cystic Fibrosis. Children’s Mercy Hospital in Kansas City, a partner of WRI, helped trial the drug. For the past seven years, WRI has given $600,000 with 100% of the funds underwiting the drug trial study at Children’s Hospital.Kalydeco represents a major treatment advance for the cystic fibrosis community. In clinical trials patients who took Kalydeco showed:

  • A significant improvement of lung function
  • A significant reduction in sweat chloride to below the diagnostic level for CF
  • Fewer pulmonary exacerbations
  • Patient-reported reduction in respiratory symptoms
  • Improvement in weight gain; and
  • No safety issues

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