Learn about cystic fibrosis, a genetic lung disorder that affects the pancreas and other organs, and how to treat and live with this chronic disease.
CF is a rare genetic disease found in about 30,000 people in the U.S. If you have CF or are considering testing for it, knowing about the role of genetics in CF can help you make informed decisions about your health care.
If you or your child has just been diagnosed with cystic fibrosis, or your doctor has recommended testing for CF, you may have many questions.
Diagnosing CF is a multistep process. A complete diagnostic evaluation should include a newborn screening, a sweat chloride test, a genetic or carrier test, and a clinical evaluation at a CF Foundation-accredited care center.
Raising a child with cystic fibrosis can bring up many questions because CF affects many aspects of your child’s life. Here you’ll find resources to help you manage your child’s daily needs and find the best possible CF care.
Living with cystic fibrosis comes with many challenges, including medical, social, and financial. By learning more about how you can manage your disease every day, you can ultimately help find a balance between your busy lifestyle and your CF care.
People with CF are living longer, healthier lives than ever before. As an adult with CF, you may reach key milestones you might not have considered. Planning for these life events requires careful thought as you make decisions that may impact your life.
People with cystic fibrosis are living longer and more fulfilling lives, thanks in part to specialized CF care and a range of treatment options.
Cystic Fibrosis Foundation-accredited care centers provide expert care and specialized disease management to people living with cystic fibrosis.
We provide funding for and accredit more than 120 care centers and 53 affiliate programs nationwide. The high quality of specialized care available throughout the care center network has led to the improved length and quality of life for people with CF.
The Cystic Fibrosis Foundation provides standard care guidelines based on the latest research, medical evidence, and consultation with experts on best practices.
As a clinician, you’re critical in helping people with CF maintain their quality of life. We’re committed to helping you partner with patients and their families by providing resources you can use to improve and continue to provide high-quality care.
As part of the Cystic Fibrosis Foundation's mission to help improve the lives of people living with cystic fibrosis, the PSDC initiative taps the CF community to inform key efforts to support the management of daily care.
Your cystic fibrosis care team includes a group of CF health care professionals who partner with you to provide specialized, comprehensive CF care.
Many cystic fibrosis patients and families face complicated issues related to getting the care they need. But CF Foundation Compass makes sure that no one has to do it alone.
For many people with cystic fibrosis, dealing with insurance is as much a part of living with the disease as nebulizers and vests. Many people with CF and their families face issues related to getting the care they need, but no one has to do it alone.
The Cystic Fibrosis Foundation is the world’s leader in the search for a cure for CF and supports a broad range of research initiatives to tackle the disease from all angles.
The CF Foundation offers a number of resources for learning about clinical trials and treatments that are being developed to improve the treatment of cystic fibrosis.
Our understanding of CF continues to evolve as scientists study what causes the disease and how it affects the body. These insights drive the development of new and better treatments and bring us one step closer to a cure.
Researchers, supported by the CF Foundation, have made tremendous advances to improve the health and quality of life of people with CF. We are committed to providing the tools and resources you need to continuously build upon this work.
Our goal is to educate policy makers about the needs of people with cystic fibrosis so that they make smart decisions about CF-related research, treatment, and access to care.
We recognize the value of tapping into the expertise that only people with CF and their families have. We invite you to share insights to help improve and develop programs and services that support the daily lives of people with CF.
Our mission is to find a cure for cystic fibrosis and improve the quality of life for those living with the disease. We can't do it alone. Help us add tomorrows by giving today.
In addition to working for a cure, the CF Foundation supports programs and policies to improve the lives of people with CF. Help us by raising awareness of CF, participating in a fundraising event, or volunteering with your local chapter.
CFTR (cystic fibrosis transmembrane conductance regulator) modulator therapies are designed to correct the function of the defective protein made by the CF gene. Because different mutations cause different defects in the protein, the medications that have been developed so far are effective only in people with specific mutations. There are currently two FDA-approved CFTR modulators: ivacaftor (Kalydeco®) and lumacaftor/ivacaftor (Orkambi®).
Ivacaftor is prescribed for people ages 2 and older who have at least one of the following mutations in the CFTR gene:
The lumacaftor/ivacaftor combination therapy is prescribed for people ages 6 and older who have two copies of the F508del mutation, which is the most common CF mutation. Several problems are caused by the F508del mutation, which prevents CFTR from achieving the correct shape and reaching the cell surface. The combination therapy works to correct this in two ways. Lumacaftor moves the defective CFTR protein to the correct place on the cell surface, and ivacaftor increases the protein's activity once it is in place.
The medications are for people with specific CF mutations. Because of this, they will not work if you do not have one or more of these mutations. The first step in knowing if either ivacaftor or the combination of lumacaftor/ivacaftor will work for you is to know what your CF mutations are. If you do not know your mutation, you can undergo genetic testing to learn what mutation or mutations you have.
The CF Foundation's Mutation Analysis Program offers free genetic testing for people with CF. Your provider must enroll you in the Mutation Analysis Program. If you are interested in enrolling, ask your provider during a care center visit.
More potential CFTR modulators are in development to address the underlying cause of the disease in people with other CF mutations.
The CFTR protein regulates the proper flow of fluids and sodium (salt) in and out of cells lining the lungs and other organs. In people with CF, mutations in the CF gene cause the protein to malfunction, or not be produced. This leads to the buildup of thick, sticky mucus, which can lead to lung infections and damage to the pancreas. It can also lead to problems in other parts of the body.
The following video shows what it looks like when the CFTR protein functions normally in the lungs -- hair-like cilia can move mucus and germs out of the airways -- and what happens when the protein doesn't work.
In people with certain mutations, CFTR modulators help the defective protein move to and function properly at the cell's surface. The following video shows how ivacaftor helps people with CF who have gating mutations. Ivacaftor binds to the defective protein at the cell surface, opens up the chloride channel and restores the proper flow of fluids and sodium.
Your care team or pharmacist will give you specific instructions for taking CFTR modulators.
For ivacaftor, adults and children ages 6 and older should take one 150 mg tablet by mouth every 12 hours with foods that contain fat, such as eggs, avocados, nuts, butter and peanut butter. Children ages 2 to 5 take a certain amount of the medication based on their weight. Typically, for children this young, the medication comes in a packet, which can be mixed into soft food or liquid and taken with foods that contain fat.
For the lumacaftor/ivacaftor combination therapy, people ages 6 and older take two tablets every 12 hours with foods that contain fat.
The effects of CFTR modulators only last for as long as the medication is in your system. Therefore, you need to take the medication every 12 hours, or as your care team recommends.
You may experience side effects when you take any medication. When discussing any new medications or changes in dosages for medications you are already taking, be sure to ask your care team about:
Let your care team know if you feel any side effect that bothers you or makes it hard for you to continue taking this medication as prescribed. Your care team can work with you to help you manage side effects or to adjust your treatment plan.
You can learn more about CFTR modulators at DailyMed, which is a service from the National Library of Medicine that provides information about drugs, including dosages and possible side effects.
Common side effects of ivacaftor include:
Common side effects of lumacaftor/ivacaftor include:
CFTR modulators are not typically available at retail pharmacies and mail-order pharmacies, but at specialty pharmacies. A specialty pharmacy fills prescriptions for drugs that are unavailable at retail pharmacies. Medications from specialty pharmacies often need to be handled and stored specially and delivered quickly.
Insurance plan coverage for CFTR modulators can vary. Check with your insurance provider to ensure that CFTR modulators are covered and what your out-of-pocket expenses may be.
You can also contact the Cystic Fibrosis Foundation Compass, a free, personalized service that can help you with insurance, financial, legal and other issues. Dedicated Compass case managers can assist in coordinating benefits or providing information about benefits offered under your plans. Contact Compass at:
Monday - Friday, 9 a.m. - 7 p.m. ETcompass@cff.org
Reference to any specific product, process or service does not necessarily constitute or imply its endorsement, recommendation or favoring by the Cystic Fibrosis Foundation. The appearance of external hyperlinks does not constitute endorsement by the Cystic Fibrosis Foundation of the linked websites, or the information, products or services contained therein.
Information contained on this site does not cover all possible uses, actions, precautions, side effects or interactions. This site is not intended as a substitute for treatment advice from a medical professional. Consult your doctor before making any changes to your treatment.
FDA-approved drug information is available at www.dailymed.nlm.nih.gov/dailymed.
Follow Us On
Help us blaze a trail to better treatments and a cure for CF. Find a clinical trial that may be right for you.
Cystic Fibrosis Foundation
4550 Montgomery Ave.
Suite 1100 N
Bethesda, MD 20814
800-344-4823 (toll free)
Sign up for our emails