Cystic Fibrosis News and Research RSS Feed - Cystic Fibrosis News and Research

Cystic fibrosis is a life threatening, inherited disease of the exocrine glands. The condition primarily affects the digestive and respiratory systems which become clogged with a thick, sticky mucus.

Cystic fibrosis is caused by a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene which results in an excess of salt and water passing into cells, causing a thick, sticky mucus to build up in bodily passageways.

In the airways, this mucus causes a persistent cough, wheezing, breathlessness, and repeated lung infections. In the digestive tract, the tubes that carry digestive enzymes from the pancreas into the small intestine become blocked, preventing the proper absorption of food nutrients. This can lead to poor weight gain, intestinal blockage (particularly in newborns), and foul-smelling, greasy stools.

There is no cure for cystic fibrosis, so treatment aims to ease symptoms and make the condition easier to live with. Bronchodilators may be used to help expand the airways, antibiotics to treat chest infections and physiotherapy can help expel mucus from the lungs.
The condition is most common in white people of northern European descent and is estimated to occurs in 1 in every 2,500 babies born in the UK, where babies are screened at birth for cystic fibrosis as part of the National Health Service newborn screening programme.
Gene therapy improves lung function in patients with cystic fibrosis

Gene therapy improves lung function in patients with cystic fibrosis

Gene therapy was shown to provide significant benefits for patients with cystic fibrosis in a recent clinical study published in The Lancet Respiratory Medicine journal. [More]
Gene therapy for cystic fibrosis shows significant benefit in lung function

Gene therapy for cystic fibrosis shows significant benefit in lung function

For the first time gene therapy for cystic fibrosis has shown a significant benefit in lung function compared with placebo, in a phase 2 randomised trial published in The Lancet Respiratory Medicine journal. The technique replaces the defective gene response for cystic fibrosis by using inhaled molecules of DNA to deliver a normal working copy of the gene to lung cells. [More]
Imperial College London researchers discover new inherited form of obesity, type 2 diabetes

Imperial College London researchers discover new inherited form of obesity, type 2 diabetes

Scientists have discovered a new inherited form of obesity and type 2 diabetes in humans. A large number of genes are involved in regulating body weight, and there are now over 30 genes known in which people with harmful changes in DNA sequence become extremely overweight. Similarly, there are a number of genes that can, when altered, cause type 2 diabetes. These conditions are inherited through families in exactly the same way as disorders such as cystic fibrosis or Huntington's disease. [More]
Study could point the way to new treatments for people with severe asthma

Study could point the way to new treatments for people with severe asthma

The immune response that occurs in patients with severe asthma is markedly different than what occurs in milder forms of the lung condition, according to researchers from the University of Pittsburgh School of Medicine. Those unique features could point the way to new treatments, they said in an article published online today in the Journal of Clinical Investigation. [More]
Spyryx receives award from Cystic Fibrosis Foundation to development therapeutic peptides for CF

Spyryx receives award from Cystic Fibrosis Foundation to development therapeutic peptides for CF

Spyryx Biosciences, Inc., a privately-held biopharmaceutical company developing novel therapeutics for obstructive lung diseases, announced today it has received an award from Cystic Fibrosis Foundation Therapeutics, which will support development of Spyryx' therapeutic peptides for cystic fibrosis (CF). [More]
Study shows cystic fibrosis is more deadly for Hispanic than non-Hispanic patients

Study shows cystic fibrosis is more deadly for Hispanic than non-Hispanic patients

Cystic fibrosis is more deadly for Hispanic than non-Hispanic patients, a disparity that is not explained by differences in their access to health care, according to a new study from the Stanford University School of Medicine. [More]
UW microbiologist receives 2015 Shaw Prize in life science and medicine

UW microbiologist receives 2015 Shaw Prize in life science and medicine

UW microbiologist Peter Greenberg has long been fascinated with how bacteria in cells communicate for cooperative activities, including the spread of infection. He started exploring the subject in 1977, thinking the implications could be far-reaching. [More]
Loyola patient celebrates 10th anniversary of heart and double-lung transplant

Loyola patient celebrates 10th anniversary of heart and double-lung transplant

On March 20, Andrew Gaumer celebrated the 10th anniversary of his life-saving heart and double-lung transplant. On Mother's Day, his wife, Andrea, gave birth to their first child, a baby boy named Jude. And on June 21, Andrew will celebrate his first Father's Day - a milestone he once feared he would never see. [More]
Pulmatrix announces new inhaled anti-infective therapy to treat CF-associated fungal infections

Pulmatrix announces new inhaled anti-infective therapy to treat CF-associated fungal infections

Pulmatrix, Inc., a clinical stage biopharmaceutical company developing innovative inhaled therapies for serious pulmonary diseases, today announced a new drug candidate, PUR1900, an inhaled anti-infective to treat fungal infections associated with cystic fibrosis (CF). [More]
Pulmatrix completes previously announced merger and closes $10 million in private placement

Pulmatrix completes previously announced merger and closes $10 million in private placement

Pulmatrix, Inc., previously known as Ruthigen, Inc., today announced the completion of its previously announced merger, effective June 15, 2015, and the closing of a private placement generating aggregate gross proceeds of $10.0 million. Participants in the private placement included pre-merger investors in Pulmatrix such as funds affiliated with 5AM Ventures, ARCH Venture Partners and Polaris Partners, as well as funds affiliated with Altitude Life Science Ventures. [More]
Pulmatrix signs ex-U.S. development agreement with Mylan for bronchodilator therapy

Pulmatrix signs ex-U.S. development agreement with Mylan for bronchodilator therapy

Pulmatrix, a clinical stage biopharmaceutical company developing innovative inhaled therapies for serious pulmonary diseases, today announced that it has entered into an ex-U.S. development agreement with Mylan N.V., one of the world's leading global pharmaceutical companies. [More]
Laurent reports positive topline results from LAU-7b Phase 1b trial in adult CF patients

Laurent reports positive topline results from LAU-7b Phase 1b trial in adult CF patients

Laurent Pharmaceuticals Inc. announced today positive topline results from a Phase 1b clinical trial with LAU-7b in adult CF patients. LAU-7b is a novel oral formulation of fenretinide, a synthetic retinoid investigated for its lipid modulating properties and potential to address the aberrant inflammatory response in CF. [More]
Study shows bacteriophages remain active against P. aeruginosa infection after nebulization

Study shows bacteriophages remain active against P. aeruginosa infection after nebulization

bacteriophage-based antibacterial therapies to treat drug resistant infections, today announced new data demonstrating that bacteriophages remain active against Pseudomonas aeruginosa (P. aeruginosa) after nebulization. [More]
Paediatric outpatient study compares effectiveness of three alternative treatments for type 1 diabetes

Paediatric outpatient study compares effectiveness of three alternative treatments for type 1 diabetes

A Montréal research team, co-supervised by Dr. Rémi Rabasa-Lhoret from the IRCM and Dr. Laurent Legault from the Montreal Children's Hospital, undertook the first paediatric outpatient study to compare three alternative treatments for type 1 diabetes. [More]
Celtaxsys closes $40M Series D financing and receives $5M CFFT grant to conduct CTX-4430 Phase 2 trial

Celtaxsys closes $40M Series D financing and receives $5M CFFT grant to conduct CTX-4430 Phase 2 trial

Celtaxsys, Inc., a clinical stage drug development company focused on developing novel therapies for inflammatory diseases, today announced the successful completion of a $40M Series D financing led by Domain Partners VIII. Additional investors in the financing include Lumira Capital, RMI Partners, Masters Capital Management and the Georgia Research Alliance Venture Fund. [More]
Non-invasive prenatal testing for Down's syndrome acceptable to parents

Non-invasive prenatal testing for Down's syndrome acceptable to parents

Non-invasive prenatal testing (NIPT) for Down's syndrome is feasible, acceptable to parents, and could be introduced into the National Health Service, UK researchers say. The results of a National Institute for Health Research study carried out by the first NHS laboratory to provide NIPT testing will be reported to the annual conference of the European Society of Human Genetics today (Saturday). [More]
UT Southwestern Medical Center Performs its 500th Lung Transplant on Cystic Fibrosis Patient

UT Southwestern Medical Center Performs its 500th Lung Transplant on Cystic Fibrosis Patient

A lung transplant to relieve Christopher Bryant Vera from devastating effects of cystic fibrosis marked the 500th lung transplant performed at UT Southwestern Medical Center, positioning the Center among an elite group of fewer than 25 in the country to achieve that benchmark. [More]
Researchers uncover critical role for two proteins in chromatin structure

Researchers uncover critical role for two proteins in chromatin structure

A team of researchers at the IRCM led by François Robert, PhD, uncovered a critical role for two proteins in chromatin structure. Their breakthrough, recently published in the scientific journal Molecular Cell, helps explain how DNA is organized in our cells. This discovery could lead to a better understanding of what causes certain types of cancer, such as lymphoma. [More]

Airway granulocytes potential CF biomarker for fungal lung disease

Researchers have found that accumulation in the airways of CXCR4+ granulocytes is associated with chronic colonisation by the fungus Aspergillus fumigatus. [More]
Enterprise Therapeutics secures additional funding to accelerate drug discovery for treatment of respiratory diseases

Enterprise Therapeutics secures additional funding to accelerate drug discovery for treatment of respiratory diseases

ENTERPRISE THERAPEUTICS Ltd, a drug discovery company focused on new therapeutics for respiratory diseases, today announced that it has secured an additional £2.4m ($3.6m) in Series-A financing. [More]
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