Buongiorno, ho appena ricevuto il seguente messaggio e mi chiedo quando il farmaco Kalydeco sarà messo in commercio. Se non erro, questo farmaco potrebbe agire anche sulla mutazione DF508.
“From: “Robert J. Beall, Ph.D., CF Foundation” Date: Tue, 31 Jan 2012 12:47:35 -0500 Subject: Important: Groundbreaking New Drug Approved for CF Dear Friend, We took a major step forward in our work to find a cure for cystic fibrosis today. This morning, the Food and Drug Administration announced its approval of a breakthrough new treatment for cystic fibrosis called KalydecoTM (previously known as VX-770). This drug is a breakthrough because it is the first to address the underlying cause of cystic fibrosis. Kalydeco works to correct a specific CF mutation, G551D, which is present in about 4 percent of people with CF in the United States. By attacking this disease-causing mutation at its core, the drug dramatically improves lung function, lowers sweat chloride levels and helps patients gain the weight they need to stay healthy. Not only is Kalydeco a major treatment breakthrough for people with the G551D mutation of CF, the science behind the drug has opened exciting new doors to research and development that may eventually lead to a cure for all people living with CF. This breakthrough drug is the result of a decade-long collaboration between the Cystic Fibrosis Foundation and Vertex Pharmaceuticals. Please know that this tremendous milestone would not have been possible without you. With your continued support, we will build on this achievement to develop new treatments that address the underlying cause of the disease for all people with cystic fibrosis. We will not rest until we find a cure. To learn more about Kalydeco, click here. http://llists.cff.org/t/123537/14748985/988/0/ Sincerely, Robert J. Beall, Ph.D. President and CEO Cystic Fibrosis Foundation 800-FIGHT-CF info@cff.org www.cff.org”