CFTR Modulator Therapies: game-changers for CF treatment
Cystic Fibrosis Awareness Month
Cystic Fibrosis affects more than 30,000 children and adults in the U.S. According to the Cystic Fibrosis Foundation, “people with CF have mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause the CFTR protein to become dysfunctional.” Today, there are several treatment options that focus on the CFTR protein known as CFTR modulator therapies. These therapies have brought life-changing opportunities to the patients who can take them.
What is the CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) protein and how does it affect those with Cystic Fibrosis (CF)?
The CFTR protein is responsible for the ongoing flow of water and chloride in and out of cells lining the lungs and other organs. When this protein isn’t operating correctly, a huge buildup of thick and sticky mucus occurs. This leads to infections in the lung, can damage the pancreas, and can also lead to problems in other parts of the body. People with CF experience breathing and digestive problems due to this thick and sticky mucus. Mutations of the different types of CFTR genes are the underlying cause of CF.
What are CFTR modulator therapies?
CFTR modulator therapies are intended to correct the malfunctioning protein made by the CFTR gene. There are currently four different CFTR modulators that target certain gene mutations: Kalydeco®, Orkambi®, Symdeko®, and Trikafta®. Currently, there are more potential CFTR modulators in development to address the underlying cause of the disease in people with other CF mutations.
April – A Trikafta® Success Story
April is a 28 year old woman who lives with Cystic Fibrosis and has the F508del mutation, which qualified her for Trikafta®. She says “I’ve basically waited my entire life for something like this and when I found out I was eligible I was speechless and my entire family cried”. The biggest change she saw was in her lung function and claims it is the highest it’s been in a while! She’s noticed improvements in her respiratory symptoms and the medication also helped her gain the weight she needed to. April loves to be outside, participate in archery, and go to the gym so this medication enabled her to get out and do these things more often! Watch April’s testimonial on Trikafta’s site.
We specialize in CF and other rare diseases
Maxor Specialty is a limited distribution provider of these four CFTR modulator therapies and other CF treatment medications. We collaborate with each point in the health system to provide better outcomes by working directly with physicians to coordinate all aspects of patient care including refill reminders and education. Our proactive, personalized care coordination has led to best in case adherence rates and improved patient quality of life. For more information on how we help our CF patients with these medications, please contact Bill Aikins at [email protected].