While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined ...

In patients with cystic fibrosis homozygous for F508del-cystic fibrosis transmembrane conductance regulator (CFTR), treatment with lumacaftor and ivacaftor significantly improved lung function ...

Cystic fibrosis (CF), the most common lethal monogenic disease in Caucasians, is characterized by recurrent bacterial infections and colonization, mainly by Pseudomonas aeruginosa, resulting in ...

- Study is Evaluating N91115 in Patients Homozygous for the F508del-CFTR Mutation and Being Treated with Orkambi™ - “Cavosonstat” Approved as the Unique Non-proprietary or Generic Name for N91115 - ...

Please provide your email address to receive an email when new articles are posted on . Combination therapy with lumacaftor/ivacaftor was safe and well tolerated for up to 120 weeks in children aged 6 ...

- For the first time, approximately 6,000 patients with one minimal function mutation and one F508del mutation have a medicine to treat the underlying cause of their disease– -12,000 people with one ...

BOSTON, Dec. 17, 2019 /PRNewswire/ -- Proteostasis Therapeutics, Inc. (NASDAQ: PTI), a clinical stage biopharmaceutical company dedicated to the discovery and development of groundbreaking therapies ...

The absence of working CFTR proteins results in poor flow of salt and water into and out of the cell in a number of organs, including the lungs. In people with the most common mutation in the CFTR ...

Vanzacaftor-tezacaftor-deutivacaftor (Vnz-Tez-Diva) can be used as an option to treat cystic fibrosis in people 6 years and over who have at least 1 F508del mutation in the cystic fibrosis ...

- New indication includes people ages 12 years and older who have one copy of the F508del mutation regardless of the other mutation type - - People with gating (F/G) or residual function (F/RF) ...