Cystic Fibrosis affects 30,000 people in the U.S. It causes an imbalance of salt in the body, which affects the pancreas shortly after birth, impedes proper digestion, and causes airways to fill with mucus, leaving patients vulnerable to lung infections and other problems. Patients are often small and malnourished and tend to have overly salty skin due to sweat glands not working properly. Currently, those diagnosed with cystic fibrosis have an average life expectancy of 37 years, but that could change for some patients with a new drug called Kalydeco (generic name ivacaftor), which has just been approved by the Food and Drug Administration (FDA). Kalydeco targets a genetic mutation in four to five percent of people with cystic fibrosis. It helps to keep the chloride gateway open in cells, resulting in a restored salt balance in the body and a prevention of future lung disease. This is the first time a drug is aiming at the underlying problem that causes cystic fibrosis, not simply treating the symptoms. The FDA fast-tracked Kalydeco and approved it in only three months, given its early signs of promise in clinical testing. Lindsay Shipp is a 27-year-old in San Diego, CA, who participated in a closely watched clinical trial for this experimental drug. She loves singing and dancing and dreamed of auditioning on Broadway until her lungs became so bad that she had to give it up. When Shipp took ivacaftor, her lungs cleared to the point that her physician told her during a...

New CF drug Kalydeco, targeting a specific genetic mutation, may increase life quality and expectancy for patients, showing early promise.

Cystic Fibrosis Drug Kalydeco Shows Promise

Cystic Fibrosis affects 30,000 people in the U.S. It causes an imbalance of salt in the body, which affects the pancreas shortly after birth, impedes proper digestion, and causes airways to fill with mucus, leaving patients vulnerable to lung infections and other problems. Patients are often small and malnourished and tend to have overly salty skin due to sweat glands not working properly. Currently, those diagnosed with cystic fibrosis have an average life expectancy of 37 years, but that could change for some patients with a new drug called Kalydeco (generic name ivacaftor), which has just been approved by the Food and Drug Administration (FDA). Kalydeco targets a genetic mutation in four to five percent of people with cystic fibrosis. It helps to keep the chloride gateway open in cells, resulting in a restored salt balance in the body and a prevention of future lung disease. This is the first time a drug is aiming at the underlying problem that causes cystic fibrosis, not simply treating the symptoms. The FDA fast-tracked Kalydeco and approved it in only three months, given its early signs of promise in clinical testing. Lindsay Shipp is a 27-year-old in San Diego, CA, who participated in a closely watched clinical trial for this experimental drug. She loves singing and dancing and dreamed of auditioning on Broadway until her lungs became so bad that she had to give it up. When Shipp took ivacaftor, her lungs cleared to the point that her physician told her during a...

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Cystic Fibrosis Drug Kalydeco Shows Promise

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