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.
Finding may help advance experimental approach to improving public health

Finding may help advance experimental approach to improving public health

Scientists have discovered a new way to manipulate how cells function, a finding that might help advance an experimental approach to improving public health: DNA vaccines, which could be more efficient, less expensive and easier to store than traditional vaccines. [More]
UMass Amherst biologist partners with Chinese scientist to develop novel drug platform

UMass Amherst biologist partners with Chinese scientist to develop novel drug platform

Margaret Riley, an evolutionary biologist at the University of Massachusetts Amherst and pioneer in fighting antibiotic-resistant bacteria, announced this week that she is partnering with a Chinese scientist to develop a new drug platform, pheromonicins. The Chinese government is committing $400 million per year to support the newly created Pheromonicin Institute of Beijing. [More]
Garlic compound can help fight lung infections in people with cystic fibrosis

Garlic compound can help fight lung infections in people with cystic fibrosis

A chemical found in garlic can kill bacteria that cause life-threatening lung infections in people with cystic fibrosis, research suggests. [More]
N30 Pharmaceuticals becomes Nivalis Therapeutics

N30 Pharmaceuticals becomes Nivalis Therapeutics

N30 Pharmaceuticals, Inc., a clinical-stage pharmaceutical company focused on the development of product candidates for cystic fibrosis (CF), announced today that the Company has changed its name to Nivalis Therapeutics, Inc. The Company's lead product candidate, N91115, is a novel inhibitor of S-nitrosoglutathione reductase (GSNOR). [More]
Finding opens avenues of research toward precision treatments to boost life expectancy for CF patients

Finding opens avenues of research toward precision treatments to boost life expectancy for CF patients

Mutation of one gene is all it takes to get cystic fibrosis (CF), but disease severity depends on many other genes and proteins. For the first time, researchers at the UNC School of Medicine have identified genetic pathways - or clusters of genes - that play major roles in why one person with CF might never experience the worse kinds of symptoms while another person will battle severe airway infection for a lifetime. [More]
Possible cause of liver disease identified in adolescents with cystic fibrosis

Possible cause of liver disease identified in adolescents with cystic fibrosis

A professor at the University of Colorado School of Medicine at the Anschutz Medical Campus and his colleagues have found a possible cause of liver disease in adolescents with cystic fibrosis. [More]
World class drug discovery team launches new company to tackle chronic respiratory disease

World class drug discovery team launches new company to tackle chronic respiratory disease

Respiratory diseases including cystic fibrosis, chronic obstructive pulmonary disease (COPD) and severe asthma represent a major unmet medical need and a significant health burden in both the developed and developing world. [More]
Arcturus to exhibit new mRNA therapeutics pipeline at two healthcare conferences

Arcturus to exhibit new mRNA therapeutics pipeline at two healthcare conferences

Arcturus Therapeutics Inc., developing the 'next wave' of RNA medicines, today announced that Joseph Payne, President and Chief Executive Officer, is scheduled to present a corporate overview, including the company's new messenger RNA (mRNA) therapeutics pipeline, at two upcoming healthcare conferences. [More]
Anthera Pharmaceuticals to host Lupus symposium in San Francisco

Anthera Pharmaceuticals to host Lupus symposium in San Francisco

Anthera Pharmaceuticals, Inc. today announced that it is inviting its shareholders and investors to a symposium on Systemic Lupus Erythematosus (SLE) at 3:30 PM on Wednesday, January 14th at the Mystic Hotel in San Francisco, CA. [More]
PTC begins rolling NDA submission for Translarna to treat nmDMD

PTC begins rolling NDA submission for Translarna to treat nmDMD

PTC Therapeutics, Inc. today announced that it has commenced a rolling submission of a New Drug Application to the United States Food and Drug Administration for Translarna™ for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). [More]
Ganciclovir drug resistance may occur more frequently in cystic fibrosis patients

Ganciclovir drug resistance may occur more frequently in cystic fibrosis patients

A drug called ganciclovir is given to lung transplant patients to protect against a life-threatening virus that is common after transplantation. [More]

CF patients require close monitoring for resistance to ganciclovir after lung transplantation

A drug called ganciclovir is given to lung transplant patients to protect against a life-threatening virus that is common after transplantation. [More]
IRCM researchers uncover mechanism that regulates dopamine levels in the brain

IRCM researchers uncover mechanism that regulates dopamine levels in the brain

Researchers in Montréal led by Jacques Drouin, D.Sc., uncovered a mechanism regulating dopamine levels in the brain by working on a mouse model of late onset Parkinson's disease. [More]

Chemical chaperone: A new therapeutic option for epilepsy

Researchers found out that the conformational defect in a specific protein causes Autosomal Dominant Lateral Temporal Lobe Epilepsy (ADLTE) which is a form of familial epilepsy. They showed that treatment with chemical corrector called "chemical chaperone" ameliorates increased seizure susceptibility in a mouse model of human epilepsy by correcting the conformational defect. [More]
Drug proves effective at inhibiting growth of drug-resistant bacteria

Drug proves effective at inhibiting growth of drug-resistant bacteria

A treatment pioneered at the University of Pittsburgh Center for Vaccine Research is far more effective than traditional antibiotics at inhibiting the growth of drug-resistant bacteria, including so-called "superbugs" resistant to almost all existing antibiotics, which plague hospitals and nursing homes. [More]
Study sheds new light on well-known mechanism required for immune response

Study sheds new light on well-known mechanism required for immune response

A new study published in the scientific journal Proceedings of the National Academy of Sciences of the United States of America sheds new light on a well-known mechanism required for the immune response. Researchers at the IRCM, led by Tarik Möröy, PhD, identified a protein that controls the activity of the p53 tumour suppressor protein known as the "guardian of the genome". [More]
PulmoFlow's nebulized drug and device combination gets FDA approval for cystic fibrosis

PulmoFlow's nebulized drug and device combination gets FDA approval for cystic fibrosis

On Tuesday, the FDA granted final approval of PulmoFlow, Inc.'s New Drug Application for Kitabis Pak – a co-packaging of generic tobramycin inhalation solution with a PARI LC PLUS Nebulizer. This is the first nebulized drug and device combination to be approved for patients with cystic fibrosis. [More]
FDA takes immediate steps to help reduce risk of spreading unsuspected uterine cancer

FDA takes immediate steps to help reduce risk of spreading unsuspected uterine cancer

The U.S. Food and Drug Administration is taking immediate steps to help reduce the risk of spreading unsuspected cancer in women being treated for uterine fibroids, which is in keeping with Kalorama Information's view that in the female health market there is much room for improvement both in the development of products and the treatment of diseases. [More]
Bayer's Amikacin Inhale and Ciprofloxacin DPI receive QIDP designation from FDA

Bayer's Amikacin Inhale and Ciprofloxacin DPI receive QIDP designation from FDA

The U.S. Food and Drug Administration has granted qualified infectious disease product (QIDP) designation to two Bayer investigational agents, Amikacin Inhale and Ciprofloxacin Dry Powder for Inhalation (DPI). [More]
Unique ability helps prolific bacterium to afflict humans, animals and even plants

Unique ability helps prolific bacterium to afflict humans, animals and even plants

New research has found that one of the world's most prolific bacteria manages to afflict humans, animals and even plants by way of a mechanism not before seen in any infectious microorganism -- a sense of touch. This unique ability helps make the bacteria Pseudomonas aeruginosa ubiquitous, but it also might leave these antibiotic-resistant organisms vulnerable to a new form of treatment. [More]