Charles Johnson, VP of Global Medical Affairs at Vertex Pharmaceuticals, presented at World Orphan Drug Congress USA 2013 on the topic, ‘Identifying biomarkers to articulate the value of an orphan drug: a look into Vertex’s Kalydeco case.’
• The first and only FDA approved drug to target the defective CFTR protein
• Approaches and strategies to deploy biomarkers research in the orphan drug discovery process
World Orphan Drug Congress USA is the largest international, commercially-focused event for the advancement of rare disease research and orphan drug development.
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