Sunday, October 30, 2011
Thursday, October 27, 2011
We don't know that what may seem like useless detours are where we might meet the most amazing people of our lives, or enjoy the best scenery, or learn the most important things.
Sometimes we fail to enjoy the trip because we are so hung up on wanting to see the itinerary. You will have the best times of your life when you just TRUST that you are exactly where you are supposed to be, doing exactly what you are meant to be doing, and that tomorrow you will be lead to the next destination.
You always have been, you always will be.
Remember to travel light, only take what you need with you. It will make things so much easier.
ENJOY YOUR FLIGHT, little birdie!
Wednesday, October 26, 2011
Friday, October 21, 2011
What if many people with cystic fibrosis lost the ability to go to their care center? What if they could no longer afford their treatments? We need your help to make sure that the thousands of people with CF who rely on Medicaid never have to answer these questions.
Twelve members of Congress could limit the ability of many in the Medicaid program to access their care. Your voice can help ensure that doesn’t happen.
This summer, Congress created a new deficit reduction “supercommittee.” Its 12 members were given broad powers to change the Medicaid program.
Medicaid provides health coverage for children and adults with CF who cannot afford other types of insurance. It is often the last resort to make sure they are able go to a CF care center, see a doctor with expertise in CF and afford their inhaled antibiotics, nebulizers, enzymes and other treatments.
Supercommittee members need to cut the country’s budget deficit, but it’s up to us to make sure they protect access to the specialized care people with CF need to stay healthy.
Help spread the word:
Not on Twitter? No problem!
Thank you for all that you do! Together, we are making a difference in the lives of people with CF.
Wednesday, October 19, 2011
Vertex Submits Application to FDA for Approval of VX-770 – First Potential Drug to Target Underlying Cause of Cystic Fibrosis
October 19, 2011
Vertex Pharmaceuticals, Inc., announced today it has submitted an application to the U.S. Food and Drug Administration for a potential new CF therapy, VX-770 — under its new proposed trade name, KALYDECO™.
If approved, it will be the first drug on the market that targets the underlying cause of cystic fibrosis. Therapies available to people with CF to date only treat symptoms of the disease.
The company is seeking approval for the drug in people with cystic fibrosis age 6 and older who carry at least one copy of the G551D mutation of cystic fibrosis.
KALYDECO (kuh-LYE-deh-koh) was discovered in a collaboration between Vertex and the Cystic Fibrosis Foundation, which provided substantial scientific, financial and clinical support throughout the development process.
“The CF Foundation is thrilled that KALYDECO is on track for possible FDA approval in 2012,” said Robert J. Beall, Ph.D., President and CEO of the CF Foundation. “This is a significant step forward in our collaboration with Vertex and is further validation of the CF Foundation’s drug development strategy. We remain committed to accelerating the development of similar targeted medicines that will benefit all people with cystic fibrosis.”
Vertex has asked the FDA for priority review of the potential drug, which, if granted, could shorten the review from 10 to 6 months. The FDA grants priority review status for several reasons, including in situations where a potential drug is considered a major treatment advance.
Results released earlier this year from Phase 3 clinical trials of KALYDECO in people with the G551D mutation of CF showed that those receiving the drug had remarkable and sustained improvements in lung function and other key symptoms of the disease, compared with those on placebo.
As FDA review of the potential drug gets underway, Vertex has set up a program to provide KALYDECO to people age 6 and older with the G551D mutation who are in critical medical need and could benefit from the treatment prior to potential approval.
The expanded access program is designed for people with CF who have highly limited lung function and meet other criteria. (Information about the program is available at CF Foundation-accredited care centers.)
KALYDECO is currently being evaluated in combination with another oral drug in development, VX-809, in people with the most common mutation of CF, Delta F508.
Vertex plans to begin the second part of the Phase 2 KALYDECO and VX-809 clinical trial this month and will evaluate the two drugs over a longer period of time.
Tuesday, October 18, 2011
"Our goals are simple and terrible: to help our children live with minimal discomfort and maximum dignity. We will not launch our children into a bright and promising future, but see them into early graves. We will prepare to lose them and then, impossibly, to live on after that gutting loss. This requires a new ferocity, a new way of thinking, a new animal. We are dragon parents: fierce and loyal and loving as hell. Our experiences have taught us how to parent for the here and now, for the sake of parenting, for the humanity implicit in the act itself, though this runs counter to traditional wisdom and advice."
Monday, October 17, 2011
Wednesday, October 12, 2011
Thursday, October 6, 2011
My name is Erin Moore, and I have an 18mo old son who has Cystic Fibrosis. Several months ago, I volunteered to work as the State Advocacy Chair, helping to raise awareness of the disease and lead the Foundation’s efforts by building relationships with targeted Congressional offices in order to increase support for the Foundation’s policy agenda. I have asked my friends and neighbors to advocate with me, and today I'm asking for your support as well.
A few weeks ago, I had the opportunity to meet with Congresswoman Jean Schmidt to discuss some of the things going on in the Cystic Fibrosis Community and how she can have a positive impact for the many people living with it. First on my list was to ask for her support in opposing cuts to the Food and Drug Administration (FDA) and the National Institute of Health (NIH). The House’s 2012 Agriculture, Food and Drug Administration Appropriations bill cuts funding for the human drugs section of the FDA by $61 million. Cuts in funding for drug evaluation in the FDA could mean fewer, more overburdened reviewers, slowing the review process. There are 8 promising CF treatments heading to the FDA for review in the next couple of years, and those with cystic fibrosis and other rare diseases can’t afford to wait for urgently needed new medications. The National Institute of Health provides funding for the research that is going on at places like Cincinnati Children's Hospital. Cuts to this funding could mean that this research must be put on hold, and we don't have time to wait.
Another item on the Policy Agenda of the Cystic Fibrosis Foundation that I was able to discuss with Congresswoman Schmidt was the importance of assuring that people with cystic fibrosis receive access to the care and treatment they need to help them live longer and healthier lives. More specifically, I was asking for Congress to help people with CF to have access to high-quality health care that adheres to the current standards recommended by CF treatment and research experts and to protect the ability of cystic fibrosis patients to get the medical care they need by reducing the increasingly prohibitive cost-share burden of this expensive disease.
Congress is currently working on the 2012 budget and many parts of that budget will impact the CF community. Congress has signaled that they are looking to reduce spending. Spending cuts that affect the Food & Drug Administration (FDA), the National Institute of Healthy (NIH) or Medicaid could be harmful to those of us dealing with CF. We need to be the voice that collectively tells Congress why preserving strong funding to these programs is so vital to the health of our community.
WHAT YOU CAN DO TO HELP!
In October, the Joint Select Committee on Deficit Reduction will be responsible for finding $1.5 trillion in savings from the Federal Budget. Even though everything in the budget (including FDA and NIH funding) will be on the table in their discussions, conversations with leaders on Capitol Hill have led us to believe that Medicaid is in the greatest danger of significant changes that could leave thousands of people with CF without vital health coverage. As a result, we think our collective voices will be most effective if we focus exclusively on Medicaid in our messages to this committee.
Contacting the members of this specific committee will be critical over the next several months if we want them to consider the very serious implications that cuts to Medicaid will have to people living with Cystic Fibrosis. Please take just a moment to click on this link to let them know that you oppose these cuts - http://bit.ly/ngfEuP
Additionally, advocacy will continue to be an important part of preserving the quality of life for Cystic Fibrosis patients. Sign up to be an advocate and let your voice be heard in Congress for yourself or your loved ones living with Cystic Fibrosis. It takes no more than a few minutes a month but has an immeasurable impact. Visit http://www.cff.org/GetInvolved/Advocate/ to sign up today.
- Posted using BlogPress from my iPad