We Must Pay Closer Attention to the Global CF Population
Based on the stringent qualifications the Cystic Fibrosis Foundation uses to determine who has cystic fibrosis, approximately 70,000...
Symdeko Approved in Australia for CF Patients Age 12 or Older With Certain Gene Mutations
Cystic fibrosis (CF) patients in Australia age 12 and older, who have two copies of the F508del mutation in the CFTR gene (a copy...
New Gene Therapy Strategy May Reverse Effects of Disease-related Mutations, Study Shows
A new strategy to genetically prevent disease-related mutations from occurring shows the potential to treat several genetic diseases,...
Eluforsen Improves CFTR Protein Function in CF Patients with F508del Mutations, Phase 1 Trial Shows
An exploratory Phase 1 trial showed that repeated intranasal treatment with the therapy eluforsen improved CFTR protein activity in...
Specific Sweat Test Better Detects Kalydeco’s Effects on CFTR Function, Study Suggests
The effects of CFTR modulator therapies such as Kalydeco (ivacaftor) can be more reliably assessed using a test that specifically...
New cystic fibrosis drugs significantly improve lung function
A triple drug combination for treating the underlying cause of cystic fibrosis is currently under study. The preliminary results show a...
Worse CF Lung Outcomes Linked with Mutations Causing Iron Overload Disorder, Study Finds
Patients with cystic fibrosis (CF) who carry certain mutations in the HFE gene, which are also behind an iron overload disorder, have...
Vertex and Treating CF: 3 Disease-modifying Therapies Make Leap from Lab to Patients
Editor’s Note: This is the second of a four-part series looking at Vertex Pharmaceuticals and its “all in for CF” approach. Here, we...
Discovering the ancient origin of cystic fibrosis, the most common genetic disease in Caucasians
Imagine the thrill of discovery when more than 10 years of research on the origin of a common genetic disease, cystic fibrosis (CF),...
Specific Gene Mutation Causes Severe CF in Georgian Jewish Patients, Study Reports
The Q359K/T360K gene mutation, which is commonly found in Jewish patients of Georgian descent, is associated with a particularly severe...
