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…
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Cambridge, Massachusetts based Vertex Pharmaceuticals Incorporated reports that the European Commission (EC) has approved expansion of the indication for its cystic fibrosis drug KALYDECO (ivacaftor), which now includes children aged two to five with cystic fibrosis (CF) and who have one of nine gating mutations in the cystic…
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…
Encouraging results of a study presented by the Katholieke Universiteit (KU) Leuven Laboratory for Molecular Virology and Gene Therapy at Leuven, Belgium indicate that improved gene therapy treatment can cure mice with cystic fibrosis (CF). The researchers note that cell cultures from CF patients also respond well to the…
The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for AB569 from Arch Biopartners Inc. as a treatment for Pseudomonas aeruginosa (P. aeruginosa) pulmonary infections in patients with cystic fibrosis. The Orphan Drug Designation is a crucial step for the development of new medicines for…
A new study entitled “Lung Microbiota Changes Associated with Chronic Pseudomonas aeruginosa Lung Infection and the Impact of Intravenous Colistimethate Sodium,” published in the PLOS One journal, highlights the potentially positive impact of intravenous antibiotic colistimethate sodium in the proportion of Pseudomonadales relative to other Gram-negative…
A new study entitled “Respiratory microbiota resistance and resilience to pulmonary exacerbation and subsequent antimicrobial intervention,” published in The International Society for Microbial Ecology Journal, investigated how bacterial communities in the lung change over time in relation to the disease state and progression of…
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…
A new study revealed that the cystic fibrosis transmembrane conductance regulator (CFTR) plays an important role in regulating the intestinal inflammatory responses. The study, entitled “CFTR Knockdown induces pro inflammatory changes in intestinal epithelial cells,” was published in the Journal of Inflammation…
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