Expert opinion on investigational drugs
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Expert Opin Investig Drugs · Mar 2011
Comment Multicenter Study Controlled Clinical TrialCystic fibrosis transmembrane regulator potentiators as promising cystic fibrosis therapies.
Several types of mutations in the cystic fibrosis transmembrane regulator (CFTR) gene lead to abnormal CFTR protein and alterations of chloride and sodium transmembrane transportation in cystic fibrosis (CF). Some investigational compounds such as VX-770 can improve CFTR protein function. ⋯ VX-770 is able to improve chloride and sodium transportation and has a good safety profile. Although such compounds have limited therapeutic targeting potential, preliminary results show great promise in the context of CF therapy.
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Expert Opin Investig Drugs · Mar 2011
ReviewThe use of vasopressin receptor antagonists in hyponatremia.
Considerable data have recently characterized hyponatremia as fairly common in the intensive care and general hospital settings. Moreover, mounting evidence suggests the association of mild degrees of hyponatremia with untoward neurocognitive and musculoskeletal outcomes. A key development in our ability to treat hyponatremia was the introduction and approval of aquaretics (vaptans). These vasopressin receptor antagonists work by increasing electrolyte-free water excretion and thus raising serum sodium concentration. ⋯ Vaptans are likely to play an important role in treating hyponatremia, given their clinical efficacy and tolerability. High cost remains an impediment for vaptans, and more studies are needed to further define their best use in hyponatremic patients.