"...And I know someday that it will all work out. You make me work so we can work to work it out. And I promise you kid that I give so much more than I get....I might have to wait, I'll never give up. I guess its half timing and the other half's luck....And I know that we can be so amazing. And baby, your love is gonna change me. And now I can see every possibility..."
Saturday, April 30, 2011
Thursday, April 28, 2011
Tuesday, April 26, 2011
Monday, April 25, 2011
Thursday, April 21, 2011
Tuesday, April 19, 2011
Friday, April 15, 2011
The Advocacy Chair (AC) position is a volunteer role that can have a significant impact on the way CF issues are treated in Washington. The AC spearheads the Foundation’s efforts in each state by building relationships with targeted Congressional offices in order to increase support for the Foundation’s policy agenda. Essentially the main responsibilities of the Advocacy Chairs are:
- Participate in the quarterly Advocacy Chair conference call and keep up with the regular update emails sent by the Public Policy Department (the day after calls, we always send a full recap for those who aren’t able to make it along with a list of next steps).
- Follow up on the action items covered on the call/in the emails, which usually involves regularly meeting and otherwise communicating with Congressional offices about the Foundation’s current policy priorities.
- Recruit 5-10 Network Advocates (usually friends, family members, or others in the CF community) in the local area.
- Use the tools and information the Public Policy department provides in order to facilitate and direct the efforts of these Network Advocates to attend meetings, call andwrite Congressional offices, and otherwise support the efforts of the Advocacy Chair.
Wednesday, April 13, 2011
Dr. Karp went on to discuss the association between inflammation and infection in a "which came first, the chicken or the egg" type presentation. I asked Dr. Burns (Drew's doc) about this after the presentation and she said that a lot of the new research and thinking around CF is to treat the inflammation to prevent the infection vs. just the thick mucus causing the infections and the inflammation providing some extra difficulty in getting rid of it all. (I completely understand that I'm probably botching all of this, but its what I took way, and thats it).
Friday, April 8, 2011
Thursday, April 7, 2011
CF Foundation Announces Investment to Speed Development of Additional Drugs to Treat the Most Common CF Mutation
April 7, 2011
Today the Cystic Fibrosis Foundation announced the expansion of its collaboration with Vertex Pharmaceuticals for the discovery and development of additional drugs aimed at treating the underlying cause of cystic fibrosis.
The new program will support development of a potential new drug called VX-661, designed to treat people with the most common genetic defect in CF, the Delta F508 mutation (this is one of Drew's mutations. YAY!). Nearly 90 percent of people with CF in the United States have at least one copy of this mutation.
VX-661 is known as a “corrector” and aims to move the defective CF protein to its proper place at the cell surface. Another corrector, known as VX-809, is already in clinical trials. By developing multiple correctors, the Foundation increases the chances of bringing new therapies to the CF community as quickly as possible.
“This new agreement will further leverage the successful collaboration with Vertex to accelerate the discovery and development of new drugs to treat a wide variety of CF patients,” said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. “Given the recent announcement of promising data of other compounds in the CF pipeline, we’re optimistic that the CF Foundation is on the right path to fundamentally change the treatment of CF by targeting the cause of the disease.”
The Foundation’s investment, which will be up to $75 million over five years, will also expedite the discovery and early development of other new correctors.
“The CF Foundation is widely recognized by doctors, nurses, scientists and those with CF as a driving force in the search for new CF medicines, and we are pleased to further expand our strong collaboration with them,” said Matthew Emmens, chairman, president and chief executive officer of Vertex. “The collaboration announced today underscores our commitment to CF and accelerates our efforts to develop new medicines as quickly as possible for people with the most common type of this disease.”
With the Foundation’s expanded support, Vertex plans to begin a Phase 2 study of VX-661 by the end of 2011 and expects to enroll people with CF who have the Delta F508 mutation.
A Phase 2 clinical trial is underway to test combinations of VX-770 and VX-809 in individuals with two copies of Delta F508 mutation. Data from the first part of this trial is expected in the middle of 2011.
Read the Vertex Pharmaceuticals press release about the announcement.