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Vertex Pharmaceuticals’ Orphan Drug Kalydeco (Ivacaftor) : Science News

October 30, 2012

       Vertex Pharmaceutical’s orphan drug Kalydeco (Ivacaftor) for the treatment of Cystic Fibrosis (CF) is in the science news : 

1)  At the Hospital for Sick Children in Toronto, Canada, scientists establish that Kalydeco  works in an “unconventional way” after developing a “new experimental system that may have potential for discovering drugs that target the basic defects caused by CFTR mutations …. The system is useful for identifying compounds that interact with rare mutations such as G551D as well as the major CFTR mutant F508del”. Christine Bear and colleagues published these findings in the Journal of Biological Chemistry’s “Paper of the Week”. 

2)  A team of researchers at McGill University and the University of British Columbia (UBC) in Canada, have “uncovered a new molecule for the treatment of CF ……. Discovered the chemical from a … marine sponge from the South Pacific Ocean. The researchers found that the chemical corrects the localization and restores the function of the defective protein that causes CF.” The findings are published in the journal Chemistry and Biology

       Vertex Pharmaceuticals schedules its 3Q Financial Results Conference Call for Thursday, November 1, 2012

References October 29, 2012 article titled,”Cystic Fibrosis Fix in a Sea Sponge ?” October 29, 2012 article titled,”Canadian scientists’ insights into Kalydeco for a rare form of cystic fibrosis”.  

Copyright  © 2012-2013, Ann-Teresa Cusenza and Orphan Druganaut Blog.  All rights reserved.

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