Ivacaftor – a Breakthrough in Cystic Fibrosis Treatment

One of the implicit themes we were taught in medical school is that an understanding of the basic science of a disease informs and guides treatment of patients. The general philosophy that we absorbed was that an understanding of the molecular or cellular defects in a disease would explain the abnormal organ physiology which would explain the patient’s abnormal signs and symptoms. This understanding of the abnormality at a molecular or cellular level would also help discover medications that cor...
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