Cystic Fibrosis Genes & Gene Therapy–June 29th 13:00 (BST)

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)

For new Rhode Island firm, peptides hold hope for disease-altering cystic fibrosis treatments

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

Cystic Fibrosis: New Assay May Help Individualize Care

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 Pharma Announces Presentation of Preclinical Data at the Basic Science Meeting of the European Cystic Fibrosis Society

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 Readies Late-Stage Cystic Fibrosis Drug Studies

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

CFTR2 – a fabulous new resource on gene mutations is now available for public use!

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!