Sep. 30, 2013 — Cystic fibrosis is a lethal genetic disorder that in France affects one child per 4,500 births. An international team led by scientists at the Institut Fédératif de Recherche Necker-Enfants Malades (CNRS/Inserm/Université Paris Descartes), led by Aleksander Edelman, has recently discovered two new compounds that could be used to treat patients carrying the most common mutation. Continue reading New Compounds Display Strong Therapeutic Potential for Cystic Fibrosis
Of the over 1,900 errors already reported in the gene responsible for cystic fibrosis (CF), it is unclear how many of them actually contribute to the inherited disease. Now a team of researchers reports significant headway in figuring out which mutations are benign and which are deleterious. Continue reading Scientists pinpoint 105 additional genetic errors that cause cystic fibrosis
Vertex’s strategy in cystic fibrosis (CF) is to provide benefit to as many CF patients as possible, and to maximize the benefit for these patients, with our approved and investigational medicines. Continue reading Vertex Updates Recent Progress and Upcoming Milestones in Clinical Development Programs
A new study led by Dr Gergely Lukacs, from the Physiology Department at McGill University, in Canada, reveals new information about the disease known as cystic fibrosis. Cystic fibrosis (CF), also known as mucoviscidosis, is a life threatening disease that severely affects the digestive system and the lungs. Continue reading Novel Therapy Against Cystic Fibrosis Discovered by Researchers
The latest cystic fibrosis research summarized by the study authors. No spin, no headlines. More added daily. cfunite.org/research
The next live CFUnite event, with added opportunities for interaction, will discuss the genes and genetics of cystic fibrosis. What does dF508, G551D and all the other numbers actually mean? Continue reading Cystic Fibrosis Genes & Gene Therapy–June 29th 13:00 (BST)
It’s been nearly two and a half decades since the gene associated with the inherited disease cystic fibrosis was discovered, paving the way for disease-modifying drugs. In that time, only one such drug has been approved in the U.S. and Europe – Vertex’s Kalydeco. And that drug is only Continue reading For new Rhode Island firm, peptides hold hope for disease-altering cystic fibrosis treatments
A rapid and quantitative assay for assessing function of the CFTR gene using intestinal organoids has been developed, according to a study conducted by Johanna F. Dekkers, MS, from the University Medical Center in Utrecht, the Netherlands, and colleagues. Continue reading Cystic Fibrosis: New Assay May Help Individualize Care
N30 Pharmaceuticals, Inc., a clinical stage, biopharmaceutical company focused on the development of therapies for cystic fibrosis (CF), announced today the presentation of key preclinical data for its novel inhibitors of S-nitrosoglutathione reductase (GSNORi). The data were Continue reading N30 Pharma Announces Presentation of Preclinical Data at the Basic Science Meeting of the European Cystic Fibrosis Society
Vertex Pharmaceuticals (VRTX) announced Tuesday night the design of two phase III studies for its combination therapy to treat the most common form of cystic fibrosis. The studies will each run for six months, so results could be ready as early as the end of 2013 or during first half of 2014.
If the studies are positive, Vertex intends to file for U.S. approval in 2014. Continue reading Vertex Readies Late-Stage Cystic Fibrosis Drug Studies
Collaborators from several institutions around the world and the US Cystic Fibrosis Foundation are excited to announce that a new resource – CFTR2 – is now available for public use! This is the result of an international research collaboration to provide information about specific Continue reading CFTR2 – a fabulous new resource on gene mutations is now available for public use!