Cystic Fibrosis Further Explained by Rare Cell Type Discovery
Investigators from the Broad Institute of MIT and Harvard and Massachusetts General Hospital (MGH) have discovered a rare cell type in...
New Gene Therapy Could Treat Cystic Fibrosis With One Dose
A new partnership in the UK will develop a gene therapy for cystic fibrosis that could treat the disease with a single dose. Cystic...
Acebilustat Seen to Prevent Pulmonary Exacerbations in CF Patients in Phase 2b Trial
Topline data from a Phase 2b trial showed that the investigative therapy acebilustat taken once daily can reduce the number and frequency...
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...
Cystic Fibrosis Treatment Approved for Use in Children Aged 2 to 5 Years
Officials with the FDA have updated the label of lumacaftor/ivacaftor (Orkambi, Vertex Pharmaceuticals), approving the treatment's use in...
Vertex Pharma (VRTX) Receives European CHMP Positive Opinion for SYMKEVI for People with CF Aged 12
Vertex Pharmaceuticals (Europe) Limited (Nasdaq: VRTX), today announced that the European Medicines Agency’s (EMA) Committee for...
Gene Mutations Linked to Other Lung Diseases May Contribute to Bronchiectasis, Study Suggests
Mutations in genes linked to other lung diseases, specifically cystic fibrosis and primary ciliary dyskinesia, may also contribute to...
Vertex Anticipates Completing Enrollment of Phase 3 Trials Testing Triple Combo Therapies
Vertex Pharmaceuticals plans to conclude the enrollment of patients in its Phase 3 programs involving its triple combo therapies to treat...
CF Babies More Likely to be Born Preterm, Weigh Less, Study Finds
Babies with cystic fibrosis (CF) are more likely to be born preterm — less than 37 weeks of pregnancy — and have significantly lower...
Arcturus’ RNA Therapy for CF Shows Promise in Preclinical Studies, Company Announces
Arcturus Therapeutics’ RNA-based approach enables the production of healthy CFTR— the protein whose dysfunction is key in cystic fibrosis...
