CFTR

May 24, 2016May 24, 2016

First Patient Dosed in Phase 2 Trial of Potential CF Treatment

Cystic Fibrosis, News

Nivalis Therapeutics, Inc., announced the first dosing of a cystic fibrosis (CF) patient enrolled in its second Phase 2 clinical trial evaluating the drug N91115, a stabilizer of the cystic fibrosis transmembrane conductance regulator ... Read more

May 17, 2016May 17, 2016

Galapagos Initiates Phase 1 Study of GLPG2451 for Cystic Fibrosis

Cystic Fibrosis, News

Galapagos NV recently announced the initiation of a phase 1 study investigating its compound GLPG2451 for the treatment of cystic fibrosis (CF). The molecule is the second compound in Galapagos’ extended CF-portfolio to ... Read more

April 4, 2016April 4, 2016

Cystic Fibrosis Patients See Potentially Promising Therapies from Proteostasis Therapeutics

Cystic Fibrosis, News

Proteostasis Therapeutics — a pharmaceutical company that develops small molecule drugs for cystic fibrosis (CF) — recently announced the latest pre-clinical results from its first-in-class genotype-agnostic amplifier and a novel corrector of CFTR ... Read more

February 23, 2016February 23, 2016

New Approach to Cystic Fibrosis Targets Anomaly Protein and Naturally Occurring Substance

Cystic Fibrosis, News

A study from researchers at Eindhoven University of Technology in the Netherlands and McGill University in Montreal, Canada, has demonstrated important progress toward the development of a new drug to battle cystic fibrosis ... Read more

February 10, 2016February 18, 2016

Vertex CF Drug, Kalydeco, Rejected as Therapy for Specific Group of Mutations by FDA

Cystic Fibrosis, News

Vertex Pharmaceuticals recently received a Complete Response Letter from the U.S. Food and Drug Administration (FDA) stating that its supplemental New Drug Application (sNDA) could not be approved in its current ... Read more

January 26, 2016May 12, 2016

Preschoolers With Cystic Fibrosis Yield Promising Results With Ivacaftor

News

New research published in The Lancet Respiratory Medicine journal reports that the oral drug ivacaftor is a safe therapy and has the potential to benefit young children between the ages of ... Read more

January 21, 2016January 25, 2016

Cystic Fibrosis Treatment Granted FDA Orphan Drug Status

Cystic Fibrosis, News

Boulder, Colorado-based Nivalis Therapeutics recently announced the U.S. Food and Drug Administration (FDA) has granted the company’s lead clinical candidate, N91115, Orphan Drug Designation for the treatment of cystic fibrosis (CF). The ... Read more

December 22, 2015December 22, 2015

Cystic Fibrosis Study into Gene Regulation Hones In on CFTR Expression

Cystic Fibrosis, News

A new study in the Nucleic Acids Research journal has found that different combinations of cis-regulatory elements — DNA stretches that regulate the transcription of nearby genes — contribute to the chromatin structure ... Read more

December 1, 2015December 1, 2015

Cystic Fibrosis Study Finds a Mutated Protein Interacts in Damaging Ways

News

Researchers at The Scripps Research Institute recently found that the mutated cystic fibrosis transmembrane conductance regulator (CFTR) protein found in many cystic fibrorsis patients interacts with wrong neighboring proteins, and ... Read more

November 23, 2015November 23, 2015

PTC Therapeutics’ CF Therapy in 2nd Fully Enrolled Phase 3 Trial

Cystic Fibrosis, News

PTC Therapeutics announced that it has completed patient enrollment in its second Phase 3 clinical trial, known as ACT CF, to evaluate Translarna™ (ataluren) as treatment for patients with nonsense mutation cystic fibrosis ... Read more