Pharmalot, Pharmalittle: Vertex and Ireland battle over price of cystic fibrosis drug

There is no known risk to a sweat test. The electrode may cause a tingling or tickling sensation from the electric current, but this is not painful.

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.

Based on the current clinical studies, Alyftrek is a safe and effective treatment for people with cystic fibrosis. The Cystic Fibrosis Foundation published a CFTR modulator therapy care guideline in 2018. Alyftrek is not included in these guidelines since it was approved by the FDA after these guidelines were published.

The symptoms of cystic fibrosis vary. Some children will have symptoms at birth, while others may not have symptoms for weeks, months, or even years. The severity of symptoms also varies, with some children showing only mild digestive and lung problems and others having severe food-absorption problems and life-threatening breathing complications. The most common symptoms of cystic fibrosis are: Salty-tasting skin , which parents notice when they kiss their child Frequent coughing , wheezing , or bouts of pneumonia or sinusitis Difficulty breathing that keeps getting worse Big appetite but poor weight gain Bulky, smelly, greasy bowel movements Over time, the symptoms of cystic fibrosis can worsen and may include: Chronic productive cough, recurrent lung infections Obstructive lung disease ( emphysema ) Chronic nasal congestion and sinus infections Pancreatitis , a painful inflammation of the pancreas Liver disease Diabetes Gallstones

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.