Thursday, April 18, 2013

Celebrate the Success in Improving the Lives of Patients with Cystic Fibrosis

 

Each year the Cystic Fibrosis Foundation reaches out and has been successful in raising funds for research in cystic fibrosis to develop treatments for this terrible disease. And the progress has been enormous since 1990 in diagnosis, locating the exact gene that regulates the chloride transfer gate in the cell walls of the lungs and intestinal mucosa.

The extent of this fatal genetic flaw reaches far beyond what I can say in this blog. Cystic fibrosis kills slowly…first by starvation and wasting, and then by pulmonary insufficiency.  It is not a pleasant sight to watch a child to fail to thrive, struggle to breathe, and eventually look like a victim of famine.

There is no cure, but these successful treatments have increased the average life expectancy from 12-16 years old in 1990 to 37 in 2013. This generation has benefitted beginning in 1993 with the development of a genetically engineered enzyme that liquefies thick mucus in the lungs, and also enzymes that allow fat and proteins  to be digested and absorbed in the intestinal tract.

Cystic fibrosis is a family disease, not just by inheritance but also how it affects the finances, daily living, and resources for brothers, sisters and parents.  Untreated, it leads to a quick death, yet prevention is a daily task which can be very successful in significantly improving the quality of life for those with CF and their loved ones.

Robert J. Beall, PhD, President of the CF Foundation tell us,

“Last year was an incredible year at the CF Foundation. People like you have shown their support and dedication to finding a cure for CF. Your generous support paved the way for the groundbreaking treatment, Kalydeco™ , the first drug to treat the underlying cause of CF.


While Kalydeco is effective for about 4 percent of patients who have a specific gene mutation, this advance brings hope to all people with CF. Kalydeco has provided us with a “road map” that is already leading to the development of more promising treatments for all with the disease.


We have made real progress toward a cure, but our work is far from over.
We will not rest until a cure is found for all people with CF.
Right now, we are expanding our research partnerships with biopharmaceutical companies and laying the groundwork to develop new therapies.


As we continue to pursue every opportunity to help develop new lifesaving therapies for people with CF, it is more important than ever that patients can access these treatments. The need for our advocacy and patient assistance programs has never been greater.
To make sure that critical research and care initiatives continue we have set an ambitious goal for the 2013 Annual Fund: $2.5 million.”

Each year in localities near you there is a ‘Great Strides’ for Cystic Fibrosis Event. It is a wonderful opportunity to sponsor or be sponsored and keep fit while creating donations for the Cystic Fibrosis Foundation.  For specifics you can go to the registration page and log in.


Help make progress possible. Please join Partners in Progress.

 

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