cystic fibrosis

PTC Therapeutics Inc. has announced the completion of patient enrollment in its second Phase 3 clinical trial of the experimental cystic fibrosis therapy Translarna™. The clinical trial, named ACT CF, is a placebo controlled study designed to evaluate the efficacy of Translarna (ataluren) in patients, age 6 or older, with nonsense…

Concert Pharmaceuticals, Inc., a clinical stage pharmaceutical company that focuses on the development of new small molecule drugs through its DCE (deuterated chemical entity) Platform®, has announced the beginning of Phase 1 multiple ascending dose clinical trial with its cystic fibrosis treatment CTP-656 (deuterium-modified ivacaftor). The announcement follows recent news…

A small study by scientists in Israel indicates that treatment with ivacaftor in people with cystic fibrosis (CF) and a specific gene mutation may help with insulin secretion problems. The study, titled “CFTR potentiator therapy ameliorates impaired insulin secretion in CF patients with a gating mutation,“ appeared in the Nov. 4, 2015,…

A new study entitled “Vitamin D and Chronic Lung Colonization in Pediatric and Young Adult Cystic Fibrosis Patients” published in the Spanish-language journal Nutrición Hospitalaria, investigated vitamin D levels in the body and its association with chronic lung colonization. The study showed that vitamin D supplements did not improve…

Assistant Professor Sigrid Ladores, PhD, from the School of Nursing, University of Alabama at Birmingham, and doctoral student Leigh Ann Bray are developing a study entitled “Female Fertility in the Age of New CFTR-Modulating Drugs.” The main focus of the research study will be to assess female patients’ understanding of…

A new study revealed that treating P. aeruginosa biofilms with the antibiotic tobramycin together with mannitol — two well-known therapies in treating lung infections in CF — did not reduce overall bacterial load. The study entitled “Mannitol Does Not Enhance Tobramycin Killing of Pseudomonas aeruginosa in a Cystic Fibrosis Model System…

OrPro Therapeutics, Inc. has announced it has been granted a patent by the US Patent and Trademark Office (USPTO) for its lead candidate Theradux™, covering its unique treatment ability for cystic fibrosis (CF). The company has been developing a treatment pipeline based on thioredoxin, a naturally secreted protein with enzymatic activity known to have…

New research has revealed potential, new strategies to fight against P. aeruginosa infection and improve both survival and quality of life in patients with cystic fibrosis. The study, entitled “Normal and Cystic Fibrosis Human Bronchial Epithelial Cells Infected with Pseudomonas aeruginosa Exhibit Distinct Gene Activation Patterns” was published in the …

A new study identified a genetic element that codifies a novel iron acquisition system that plays a previously undiscovered role in P. aeruginosa airway infection during cystic fibrosis (CF). The discovery could aid in improving prevention and treatment of P. aeruginosa in CF patients. Learn more…

Contrary to previous research, in a recent study published in the journal BMC Pulmonary Medicine, scientists did not find any general elevated risk for anxiety and depression among patients with Cystic Fibrosis. In people with CF, a defective gene causes a thick buildup of mucus…