Recorded: EIP 2011 ● Language: English ● Duration: 27 minutes Topic: Pediatric Rare Diseases


Speaker: STEVE CUNNINGHAM, Consultant Respiratory Paediatrician, Department of Respiratory and Sleep Medicine, Royal Hospital for Sick Children, Edinburgh, United Kingdom

  Learning Objectives: At the end of the session participants will: (1) Gain knowledge of the current mechanisms of disease in cystic fibrosis and how therapies are being developed to interact these mechanisms (2) Develop an appreciation of how novel clinical biomarkers are required as outcomes in slowly evolving diseases (3) Understand the successes and failures of recent novel medicines for cystic fibrosis and what the future holds