HealthWACH: Miracle drug for Cystic Fibrosis

HealthWACH: Miracle drug for Cystic Fibrosis


Cystic Fibrosis a hereditary disease that affects about 300,000 children and adults in the U.S. This disease is found in the lungs and digestive system and it causes the affected organ to produce unusual sticky, thick mucus.

Eventually the mucus causes the lungs to clog and affects the pancreasâ?? ability to absorb and break down food. In earlier years, cystic fibrosis patients could expect to live until they are around 12, but now, medicine has extended lives of these patients all the way until their 40s. Cystic Fibrosis is a hereditary disease that includes a defect in a gene that changes a protein. This protein performs the movement of regulating salt in and out of cells. The disease is passed down by the childâ??s parent, but the gene has to be in both parents for the child to inherit the disease. If only one copy of the gene is present, the disease will not be passed along to the child, but if their child decides to reproduce, they may have a baby that will have cystic fibrosis. The signs of having cystic fibrosis vary depending on how severe the disease is. Most symptoms begin during infancy, but others may notice symptoms around adolescence or adulthood. Symptoms may improve or worsen in time, but normal signs of cystic fibrosis include:

  • Feeling out of breath

  • Wheezing

  • Lung infections

A new drug has been fast tracked by the FDA to help cystic fibrosis patients. Kalydeco was recently approved to treat 4 percent of cystic fibrosis patients who have a specific gene mutation. This drug is the key to allowing patients live longer and healthier lives. The fast track program decreases the time of FDA approval and allows patients to receive their medications faster. Kalydeco has helped patients with symptoms such as breathing, wheezing, and the ability to exercise.