Pharmalot, Pharmalittle: Vertex battles Ireland over the price of its cystic fibrosis drug

Your health care provider will decide the correct dose of Alyftrek based on your age and weight. Alyftrek is a tablet that is taken by mouth. It should be swallowed whole with food that contains fat, such as eggs, nuts, meats, or dairy products, to help the body absorb the medicine properly. The treatment plan usually follows these steps after receiving the recommended liver evaluations and tests: For people aged 6 to <12 years (<40 kg): Take three tablets (vanzacaftor 4 mg/tezacaftor 20 mg/ deutivacaftor 50 mg) once daily with fat-containing food. For people aged 6 to <12 years (≥40 kg) or ≥12 years: Take two tablets (vanzacaftor 10 mg/tezacaftor 50 mg/deutivacaftor 125 mg) once daily with fat-containing food. Dose adjustments may be needed in people with liver problems. Alyftrek should be taken at the same time each day to maintain steady levels of the medicine in the body. Keep all appointments with your health care provider before and while taking Alyftrek. Your health care provider may monitor how well your liver is working.

A sweat test measures the amount of chloride in your sweat . Chloride is a type of electrolyte . Electrolytes are electrically charged minerals  that help control the amount of fluids and the balance of acids and bases (pH balance) in your body. Chloride and sodium form the salt found in your sweat.

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.

Learn more from pulmonologist Sarah Chalmers, M.D. Cystic fibrosis (CF) is a condition passed down in families that causes damage to the lungs, digestive system and other organs in the body. CF affects the cells that make mucus, sweat and digestive juices. These fluids, also called secretions, are usually thin and slippery to protect the body's internal tubes and ducts and make them smooth pathways. But in people with CF, a changed gene causes the secretions to become sticky and thick. The secretions plug up pathways, especially in the lungs and pancreas. CF gets worse over time and needs daily care, but people with CF usually can attend school and work. They often have a better quality of life than people with CF had in past decades. Better screening and treatments mean that people with CF now may live into their mid- to late 50s or longer, and some are being diagnosed later in life.

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.