Vertex Outlines 2016 Business Priorities to Support the Discovery and Development of New Transformative Medicines for the Treatment of Cystic Fibrosis and Other Serious Diseases

http://investors.vrtx.com/releasedetail.cfm?ReleaseID=949601

-Approximately 25,000 people with cystic fibrosis worldwide currently eligible for treatment with ORKAMBI® (lumacaftor/ivacaftor) or KALYDECO® (ivacaftor); Vertex Continue reading Vertex Outlines 2016 Business Priorities to Support the Discovery and Development of New Transformative Medicines for the Treatment of Cystic Fibrosis and Other Serious Diseases

CF Researchers Identify Signaling Networks Controlling Mutant CFTR Protein Folding

http://cysticfibrosisnewstoday.com/2015/12/29/signaling-networks-controlling-mutant-cftr-protein-folding-identified/

 

From Cystic Fibrosis News Today
By Magdalena Kegel

A new study published in the journal e-LIFE has taken a novel approach to looking at deficits in protein folding associated with mutations in the CF transmembrane conductance regulator Continue reading CF Researchers Identify Signaling Networks Controlling Mutant CFTR Protein Folding

Top 10 Cystic Fibrosis Articles of 2015 Highlights from the past year’s work in disease treatments, therapies and some personal stories

http://cysticfibrosisnewstoday.com/2015/12/28/top-10-cystic-fibrosis-news-stories-of-2015/

Originally from Cystic Fibrosis News Today, online

Cystic Fibrosis News Today has covered news of the latest treatments, events, clinical trials, and research updates in cystic fibrosis on a daily basis throughout the past year. As 2015 comes Continue reading Top 10 Cystic Fibrosis Articles of 2015 Highlights from the past year’s work in disease treatments, therapies and some personal stories

Cystic Fibrosis Phase 2 Trial of Drug Aiming to Stabilize Key CF Gene Underway

http://cysticfibrosisnewstoday.com/2015/12/02/cystic-fibrosis-phase-2-trial-drug-aiming-stabilize-key-cf-gene-underway/

Published originally in Cystic Fibrosis News Today
By Charles Moore

Nivalis Therapeutics, Inc., a Boulder, Colorado-based clinical stage pharmaceutical company, announced that the first patient has been dosed in the Phase 2 clinical trial of its lead investigational drug, N91115, a stabilizer Continue reading Cystic Fibrosis Phase 2 Trial of Drug Aiming to Stabilize Key CF Gene Underway

LOSING TO WIN: HOW FUNGI CAUSE TROUBLE IN CYSTIC FIBROSIS

Cystic fibrosis (CF) affects about 30,000 Americans leaving them with a variety of symptoms ranging from gastrointestinal problems to the more recognized respiratory complications. The condition is caused by a mutation Continue reading LOSING TO WIN: HOW FUNGI CAUSE TROUBLE IN CYSTIC FIBROSIS

Concert Pharmaceuticals Initiates Phase 1 Multiple Ascending Dose Trial with CTP-656 for Cystic Fibrosis

Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) today announced that it has initiated a Phase 1 multiple ascending dose clinical trial with CTP-656. CTP-656 (deuterium-modified ivacaftor) is a novel, potentially disease-modifying Continue reading Concert Pharmaceuticals Initiates Phase 1 Multiple Ascending Dose Trial with CTP-656 for Cystic Fibrosis

Gene therapy: A promising candidate for cystic fibrosis treatment

An improved gene therapy treatment can cure mice with cystic fibrosis (CF). Cell cultures from CF patients, too, respond well to the treatment. Those are the encouraging results of a study presented by the Laboratory for Molecular Continue reading Gene therapy: A promising candidate for cystic fibrosis treatment

Spraying new genes into your lungs could ease misery of cystic fibrosis

For Oli Dillon, an effective therapy for cystic fibrosis can’t come soon enough. Thanks to a groundbreaking development, he’s already had a tantalising ‘taster’ of a treatment that could transform lives such as his. Continue reading Spraying new genes into your lungs could ease misery of cystic fibrosis

First steps towards new therapy for diseases caused by defective anion transport

The research, published this week in Nature Chemistry, was led by Professor Tony Davis from the School of Chemistry, and Dr David Sheppard from the School of Physiology and Pharmacology, and was supported by a grant from the Continue reading First steps towards new therapy for diseases caused by defective anion transport