Nivalis Therapeutics Announces FDA Orphan Drug Designation for N91115 in Cystic Fibrosis

https://globenewswire.com/news-release/2016/01/15/802351/0/en/Nivalis-Therapeutics-Announces-FDA-Orphan-Drug-Designation-for-N91115-in-Cystic-Fibrosis.html

Nivalis Therapeutics, Inc. (NASDAQ:NVLS), a clinical stage pharmaceutical company focused on developing innovative solutions for people with cystic fibrosis (“CF”), today announced Continue reading Nivalis Therapeutics Announces FDA Orphan Drug Designation for N91115 in Cystic Fibrosis

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

Researchers Further Illuminate Pathway for Treatment of Cystic Fibrosis

http://www.newswise.com/articles/researchers-further-illuminate-pathway-for-treatment-of-cystic-fibrosis

CHAPEL HILL, NC – It is well established that people with cystic fibrosis (CF) have two faulty copies of the CFTR gene, but debate continues on the question of whether certain symptoms of the Continue reading Researchers Further Illuminate Pathway for Treatment of Cystic Fibrosis

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

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

Vertex Pharmaceuticals Incorporated: Profit on the Horizon

Vertex Pharmaceuticals (NASDAQ:VRTX) released outstanding third-quarter earnings on Wednesday with sales of both cystic fibrosis drugs, Orkambi and Kalydeco, selling like hotcakes. Continue reading Vertex Pharmaceuticals Incorporated: Profit on the Horizon

Vertex Turns Up the Heat on Cystic Fibrosis Challengers

Back in 2012, Vertex Pharmaceuticals (NASDAQ:VRTX) changed thousands of lives when it won approval to market Kalydeco to treat cystic fibrosis patients in the U.S. and EU. Cystic fibrosis, or CF, can be caused by one of many mutations Continue reading Vertex Turns Up the Heat on Cystic Fibrosis Challengers

Cystic Fibrosis Foundation Therapeutics Announces $7.5 Million Award to Discover New Therapies for Nonsense Mutations

Southern Research will use a sophisticated screening process to identify compounds in its chemical library that could promote the production of functional CFTR protein. In individuals with CF, a defective CFTR protein leads to Continue reading Cystic Fibrosis Foundation Therapeutics Announces $7.5 Million Award to Discover New Therapies for Nonsense Mutations

Discovery of Iron Metabolism-associated Gene Could Lead to New P. aeruginosa Treatments for Cystic Fibrosis Inhibition of PA4834 could contribute to the efficient eradication of P. aeruginosa in Cystic Fibrosis patients

http://cysticfibrosisnewstoday.com/2015/10/19/discovery-iron-metabolism-associated-gene-lead-new-p-aeruginosa-treatments-cystic-fibrosis/

Originally published in Cystic Fibrosis News Today
By Ana Pamplona, PhD

A new study entitled “A novel siderophore system is essential for the growth of Pseudomonas aeruginosa in airway mucus” published in the Scientific Reports journal by researchers from the PLUS Project for Medical Science and Yonsei Continue reading Discovery of Iron Metabolism-associated Gene Could Lead to New P. aeruginosa Treatments for Cystic Fibrosis Inhibition of PA4834 could contribute to the efficient eradication of P. aeruginosa in Cystic Fibrosis patients

Galapagos advances triple combination therapy in cystic fibrosis

GLPG2665, a next-generation (C2) corrector, together with the other two components of the triple combination, show up to six-fold greater chloride transport than Orkambi1) in vitro

– Triple combination therapy of C2 with GLPG2222 and GLPG1837 expected to Continue reading Galapagos advances triple combination therapy in cystic fibrosis