Phase III Study Shows Vertex Drug Significantly Boosts Lung Function in Children with CF

You might want to take a friend or family member with you to the appointment to help you remember information. Before your appointment, make a list of: Symptoms and when they started. Include anything that makes symptoms worse or better. All medicines, vitamins, herbs and supplements that you or your child take. Include the doses. Family history, such as whether anyone in your family has cystic fibrosis. Treatment you or your child have had for CF, if any. Include what the treatment was and if it helped. Any other medical conditions and their treatments. Questions to ask your healthcare professional. Questions to ask may include: What is likely causing these symptoms? What kinds of tests are needed? What treatment do you recommend? I or my child have other health conditions. How will cystic fibrosis affect them? Are there any limits needed? Feel free to ask other questions during your appointment.

If you or your child has symptoms of cystic fibrosis — or if someone in your family has CF — talk with your healthcare professional about testing for the condition. Make an appointment with a doctor who has skills and experience in treating CF. CF requires regular follow-up with your healthcare professional, at least every three months. Call your healthcare professional if you have new or worsening symptoms, such as more mucus than usual or a change in the mucus color, lack of energy, weight loss, or severe constipation. Get medical care right away if you're coughing up blood, have chest pain or trouble breathing, or have severe stomach pain and bloating. Call 911 or your local emergency number or go to the emergency department at a hospital if: You're having a hard time catching your breath or talking. Your lips or fingernails turn blue or gray. Others notice that you're not mentally alert.

Cystic fibrosis (CF) is a genetic disorder, which means you get it from your parents at birth. It affects your lungs, pancreas, and other organs. CF changes the way chloride (salt) moves through the cells of your body. This causes the mucus (which should be thin and slippery) in various organs to become thick and sticky. Over time, this thick mucus builds up inside your airways, making it hard to breathe. The mucus traps germs and leads to infections and inflammation. It can also cause severe, long-term damage to the lungs and lead to respiratory failure (inability to breathe normally) and death. In the pancreas, the thick mucus caused by CF prevents the release of digestive enzymes when you eat. This leads to malnutrition and poor growth. CF can also cause liver disease, reproductive problems, and cystic fibrosis-related diabetes (CFRD). More than 40,000 people in the U.S. live with CF. Doctors diagnose about 1,000 new cases each year. Today, more than half of the CF population is aged 18 or older, and new treatments have expanded the life expectancy by decades.

Your health care provider will monitor your progress while you are taking Alyftrek to make sure the treatment is effective. One key test used to measure progress is lung function testing (like FEV₁%), which checks how well your lungs are working. If Alyftrek is working, your FEV₁% may get better or remain the same over time. Another important measure is sweat chloride levels. Higher sweat chloride levels are a sign of cystic fibrosis, and Alyftrek may help lower these levels by helping the CFTR protein work better. Your health care provider may order sweat chloride tests to track improvements. In addition to lab tests, you may notice fewer lung infections, better breathing, less coughing, and increased energy levels. Your health care provider will also monitor for any side effects and may adjust your treatment plan if needed. Always follow up with your health care team to discuss any changes in symptoms or concerns about your treatment. Do not stop taking Alyftrek without talking to your health care provider first. Your symptoms may get worse again if you stop or change any of your medicines.

You don't need any special preparations for a sweat test, but you should avoid applying any creams or lotions to the skin for 24 hours before the test.