August 8 2009
source Cystic Fibrosis Foundation
A story in today’s Xconomy features an interview with researcher Bonnie W. Ramsey, M.D. who has devoted her career to the study of cystic fibrosis.
Ramsey says VX-770, an oral therapy in development by Vertex Pharmaceuticals, has the potential to be one of the first drugs to treat the underlying genetic cause of CF. VX-770 is currently in pivotal Phase 3 clinical trials.
The possibility that a drug could correct the faulty protein in CF “…is such a giant step forward, it’s like a man walking on the moon,” says Ramsey of VX-770.
The drug is being developed, explains Ramsey, as a result of the CF Foundation’s strategy to find a small molecule that could correct the basic defect in CF, as well as the Foundation’s financial and scientific support of the Vertex program.
The article highlights the pioneering steps the CF Foundation took to discover the drug and launch it into the development process.
Ramsey remembers the dark days of CF when diagnosed children had a life expectancy of about 17 years, the story says. “The outlook is much brighter for patients now, as quality of life has improved significantly, and median lifespans have doubled
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