Moderna Therapeutics Inc. has emerged as a partner of choice for drug makers eager to supplement their own research.
Vertex Pharmaceuticals Inc. became the latest established biopharma company Continue reading Moderna, Vertex to collaborate on cystic fibrosis
Why did I go through the effort of finding out my CF mutations? I am a recipient of a bilateral lung transplant, I no longer have CF in my lungs and I am living post transplant now 16 years. So, what can knowing my alleles do Continue reading Mutations and What They Might Mean
Precision medicine sounds like an inarguably good thing. It begins with the observation that individuals vary in their genetic makeup and that their diseases and responses to medications differ as a result. It then aims to find the right drug, for the right patient, at the Continue reading The Paradox of Precision Medicine
By Malika Ammam, PhD
Originally posted in Cystic Fibrosis News Today, 2/19/16
Collaborative research between Eindhoven University of Technology (Netherlands), McGill University (Canada), and the University of Duisburg-Essen (Germany) has identified a promising approach that could Continue reading CF Researchers Identify Way to Move Key Mutant Protein to Its Proper Spot in Cells
Galapagos NV (Euronext & NASDAQ: GLPG) announced today the first dosing in its Phase 2 exploratory program of GLPG1837 in patients with cystic fibrosis (CF).
GLPG1837 is a candidate CFTR potentiator drug in clinical development for the treatment of Class III mutations in cystic fibrosis. The SAPHIRA Phase 2 Continue reading Galapagos starts SAPHIRA Phase 2 study with GLPG1837 in cystic fibrosis patients
Five things we need to stop calling ourselves and why.
You know what the labels are: Patient. CFer. Cystic. Fibro/Cyster. Sufferer. It makes me cringe just to write them! I strongly dislike these words, and believe that our community would be better off if they were never said again. I see Continue reading Don’t Call Me That – Language is Power. Guest blog by Chris Kvam, Esq.
Nivalis Therapeutics, Inc. announced that the FDA has granted orphan drug designation to its lead candidate, N91115, for the treatment of patients suffering from cystic fibrosis. The stock surged 15.5% on the news. Continue reading Nivalis’ N91115 Gets Orphan Drug Status for Cystic Fibrosis