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How One Family is Making a Million Dollar Difference
(a story about us)

http://www.divinecaroline.com/life-etc/culture-causes/how-one-family-making-million-dollar-difference

Woko Ad:


Video Games May Become New Treatment for Cystic Fibrosis

A game devloped by the Emergent Media Center @ Champlain College maybe the next step in helping children manage their treatment:

http://www.empowher.com/cystic-fibrosis/content/video-games-may-become-new-treatment-cystic-fibrosis

Vertex Begins Phase 3 Study of Kalydeco and VX-809 — Targeting Most Common CF Mutation
February 27, 2013

Vertex Pharmaceuticals Inc. announced the initiation of international Phase 3 clinical trials of Kalydeco™ in combination with VX-809, a CF drug in development. The combined therapies are designed to treat the underlying cause of cystic fibrosis.  
The six-month studies will examine the combination treatment in people ages 12 and older who have two copies of the Delta F508 mutation of CF, the most common mutation in cystic fibrosis. About 50 percent of people with CF in the United States have two copies of the Delta F508 mutation.
The studies will be conducted at approximately 200 clinical trial sites in North America, Europe and Australia. In total, about 1,000 patients will be enrolled across the two studies, which will test two different doses.
The U.S. Food and Drug Administration (FDA) recently awarded a “Breakthrough Therapy Designation” to Kalydeco and VX-809. This new designation is intended to speed the development of select potential therapies that treat life-threatening diseases or conditions. As a result, the timeframe of the Phase 3 trial of Kalydeco and VX-809 has been considerably shortened in order to accelerate testing. Most Phase 3 trials are about one year long.
Both Kalydeco and VX-809 are designed to treat the root cause of CF — a faulty gene and its protein product, CFTR. In January 2012, the FDA approved Kalydeco when taken alone for people with the G551D mutation of CF ages 6 and older.
People with CF and their families who have questions about the Phase 3 trial of Kalydeco and VX-809 should speak with their CF health care providers or call Vertex Medical Information at 1-877-634-8789.

Phase 2 Study of Kalydeco and VX-661 in Combination Shows Promising Results
April 18, 2013

Vertex Pharmaceuticals Inc. today announced promising results from a Phase 2 clinical trial of its cystic fibrosis drug Kalydeco™ in combination with another potential CF therapy, VX-661.
Both Kalydeco and VX-661 are designed to treat the underlying cause of CF, a faulty gene and its protein product, known as CFTR.
“Today’s announcement is more terrific news for the CF community,” said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. “These results show that another potential therapy from our ongoing collaboration with Vertex can be paired with Kalydeco and achieve promising improvements in health. The data further validate our strategy of using small molecule compounds to address the basic genetic defect in cystic fibrosis.”
The results showed a significant improvement in lung function in people with two copies of the most common CF mutation, Delta F508, who took the combination treatment, compared with those who received a placebo.
About 50 percent of people with CF in the United States have two copies of the Delta F508 mutation.
The Phase 2 study enrolled 128 people ages 18 and older with two copies of the Delta F508 mutation of CF. Four different doses of VX-661 were evaluated in combination with Kalydeco. Those who received the two highest doses of VX-661 showed the greatest improvement in lung function.
VX-661 is the second potential therapy that Vertex has studied in combination with Kalydeco in people with two copies of the Delta F508 mutation. Vertex is also studying Kalydeco with VX-809 in two Phase 3 trials that will enroll about 1,000 people at approximately 200 clinical trial sites in North America, Europe and Australia.
Vertex plans to conduct additional studies of VX-661 and Kalydeco in combination, pending discussions with regulatory agencies.
In today’s announcement, Vertex also said it plans to begin a Phase 1 study later this year to evaluate Kalydeco in combination with the potential CF drug VX-983 in people with two copies of the Delta F508 mutation.
The CF Foundation played a key role in the development of Kalydeco, VX-809, VX-661 and VX-983, providing significant scientific, clinical and financial support. The U.S. Food and Drug Administration approved Kalydeco in January 2012 for people with the G551D mutation of CF.