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Cystic Fibrosis News

FDA Approves Kalydeco (ivacaftor) for Cystic Fibrosis in Children Ages 12 to <24 Months with Certain Mutations in the CFTR Gene

Posted 3 days ago by Drugs.com

BOSTON--(BUSINESS WIRE)--Aug. 15, 2018-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) approved Kalydeco (ivacaftor) to include use in children with cystic fibrosis (CF) ages 12 to

FDA Approves Expanded Indication for Orkambi (lumacaftor/ivacaftor) in Children Ages 2-5 Years

Posted 4 days ago by Drugs.com

BOSTON--(BUSINESS WIRE)--Aug. 7, 2018-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) has approved Orkambi (lumacaftor/ivacaftor) to include use in children ages 2 through 5 years with cystic fibrosis (CF) who have two copies of the F508del-CFTR mutation, making it the first medicine approved to treat the underlying cause of CF in ...

FDA Approves Symdeko (tezacaftor/ivacaftor and ivacaftor) to Treat Cystic Fibrosis in People Ages 12 and Older with Certain Mutations in the CFTR Gene

Posted 14 Feb 2018 by Drugs.com

BOSTON--(BUSINESS WIRE) Feb 12, 2018 – Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) approved Symdeko (tezacaftor/ivacaftor and ivacaftor) for treating the underlying cause of cystic fibrosis (CF) in people ages 12 and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator ...

Kalydeco Approval Widened for More Types of Cystic Fibrosis

Posted 18 May 2017 by Drugs.com

THURSDAY, May 18, 2017 – The U.S. Food and Drug Administration says it has expanded approval for the cystic fibrosis drug Kalydeco (ivacaftor) to include 33 mutations of the disease, up from the previous 10 mutations. In a media release, the agency said its decision was based on results of laboratory testing and previous clinical trials. "Many rare cystic fibrosis mutations have such small ...

Cystic Fibrosis Survival Better in Canada Than in U.S.

Posted 14 Mar 2017 by Drugs.com

MONDAY, March 13, 2017 – People with cystic fibrosis are living longer than ever, but those in Canada live nearly 10 years longer than those in the United States, a new study finds. Cystic fibrosis is a genetic disease that affects the lungs and digestive tract. It causes the production of a thick, sticky mucus that clogs the lungs. This may cause life-threatening lung infections. People with ...

Patient Organizations Offer Advice on Reforming Obamacare

Posted 7 Mar 2017 by Drugs.com

MONDAY, March 6, 2017 – The U.S. Congress needs to focus on certain areas as it considers changes to the Affordable Care Act, according to a coalition of 11 major patient groups. "As Congress begins debate on how to improve the nation's health care system, our organizations will evaluate any proposed changes based on a set of Consensus Health Care Reform Principles we believe necessary to ...

US Families Spend 1.5 Billion Hours Yearly on Kids With Special Health Needs

Posted 27 Dec 2016 by Drugs.com

TUESDAY, Dec. 27, 2016 – Families in the United States spend 1.5 billion hours each year providing home health care to their chronically ill or disabled children, a new study shows. The hours of health care these parents and other family members provide would cost $36 billion if performed by home health workers who received the going rate – or $12 billion if unskilled workers receiving minimum ...

FDA Approves Orkambi (lumacaftor/ivacaftor) for Use in Children with Cystic Fibrosis Ages 6 through 11 who have Two Copies of the F508del Mutation

Posted 29 Sep 2016 by Drugs.com

BOSTON--(BUSINESS WIRE) September 28, 2016 – Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) approved Orkambi (lumacaftor/ivacaftor) for use in children with cystic fibrosis (CF) ages 6 through 11 who have two copies of the F508del mutation. People with this mutation represent the largest population of those with CF, a rare, ...

Why Do Some Kids Escape Terrible Genetic Disorders?

Posted 11 Apr 2016 by Drugs.com

MONDAY, April 11, 2016 – Some severe genetic birth defects, like cystic fibrosis, are considered inescapable, automatically dooming children to disease or disability if they inherit a mutated gene from their parents. But researchers now have found rare instances where children have beaten the odds and defied their genetic destiny. Analysis of nearly 600,000 people's genetic makeup revealed 13 ...

Cystic Fibrosis Drug Seems OK for Preschoolers: Study

Posted 25 Jan 2016 by Drugs.com

MONDAY, Jan. 25, 2016 – The cystic fibrosis drug ivacaftor appears safe and effective for young children, a drug company-funded study suggests. "This was a small trial, but we are thrilled to see these results," said study leader Jane Davies, from the National Heart and Lung Institute at Imperial College London in England. "Ivacaftor is a potential new treatment to offer children aged 2 years ...

Scientists, Ethicists Debate Future of Gene Editing

Posted 3 Dec 2015 by Drugs.com

WEDNESDAY, Dec. 2, 2015 – What if faulty genes in your DNA could be easily corrected, avoiding the ravages of diseases like cystic fibrosis or certain cancers? That is the promise of gene editing, a new technique being heralded as an enormous advancement in genetic engineering. Scientists say its speed, efficiency and cost-effectiveness make it an excellent tool for replacing rogue genes that ...

Orkambi Approved for Cystic Fibrosis

Posted 2 Jul 2015 by Drugs.com

THURSDAY, July 2, 2015 – Orkambi (lumacaftor and ivacaftor) has been approved by the U.S. Food and Drug Administration to treat cystic fibrosis in patients who have inherited two copies of a specific gene mutation from their parents. The F508del mutation causes production of an abnormal protein that disrupts transport of water and chloride through the body, the FDA said Thursday in a news ...

FDA Approves Orkambi (lumacaftor/ivacaftor) for Cystic Fibrosis

Posted 2 Jul 2015 by Drugs.com

July 2, 2015 – The U.S. Food and Drug Administration today approved the first drug for cystic fibrosis directed at treating the cause of the disease in people who have two copies of a specific mutation. Orkambi (lumacaftor 200 mg/ivacaftor 125 mg) is now approved to treat cystic fibrosis (CF) in patients 12 years and older, who have the F508del mutation, which causes the production of an ...

Gene Therapy for Cystic Fibrosis Shows Some Promise in Study

Posted 2 Jul 2015 by Drugs.com

THURSDAY, July 2, 2015 – Gene therapy for cystic fibrosis has for the first time shown slight but significant benefit on lung function, new British research reveals. In a randomized trial, patients inhaled molecules of DNA that aimed to replace the defective gene responsible for cystic fibrosis with a healthy, working copy of the gene in the lungs. "Patients who received the gene therapy showed ...

Cystic Fibrosis Might Be Deadlier for Hispanics, Study Suggests

Posted 30 Jun 2015 by Drugs.com

TUESDAY, June 30, 2015 – Cystic fibrosis is deadlier for Hispanic patients than others, and genetic differences may make Hispanics less likely to benefit from new treatments for the disease, researchers report. "We need to ask if the care model for patients with [cystic fibrosis] is working for this minority group," study author Dr. MyMy Buu, an instructor in pediatric pulmonary medicine at ...

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