Clinical pharmacology and therapeutics
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Clin. Pharmacol. Ther. · Jan 2017
ReviewCan Cystic Fibrosis Patients Finally Catch a Breath With Lumacaftor/Ivacaftor?
Cystic fibrosis (CF) is a life-limiting disease caused by defective or deficient cystic fibrosis transmembrane conductance regulator (CFTR) activity. The recent US Food and Drug Administration (FDA) approval of lumacaftor combined with ivacaftor (Orkambi) targets patients with the F508del-CFTR. The question remains: Is this breakthrough combination therapy the "magic-bullet" cure for the vast majority of patients with CF? This review covers the contemporary clinical and scientific knowledge-base for lumacaftor/ivacaftor and highlights the emerging issues from recent conflicting literature reports.
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This translational report on the current field of emerging cell therapy and tissue engineering therapies includes the challenges and opportunities to accelerate clinical translation in regenerative medicine. Translation of regenerative medicine refers to the transfer from bench (proof-of-concept) to bedside (clinical trials), and finally commercialization. Regenerative medicine therapies have the capacity to replace, repair, and regenerate cells, tissues, and organs to restore normal function in the body. These emerging therapies are shifting the paradigm from treatment-based to cure-based therapies.