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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.
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]
University of Missouri researchers identify key protein component that causes cystic fibrosis

University of Missouri researchers identify key protein component that causes cystic fibrosis

Nearly 70,000 people worldwide are living with cystic fibrosis, a life-threatening genetic disease. There currently is no cure for the condition, but researchers from the University of Missouri have identified a key component in the protein that causes the disease. It is a finding that may lay the foundation for the development of new medications and improved therapies. [More]
Two-drug combination improves lung function in some cystic fibrosis patients

Two-drug combination improves lung function in some cystic fibrosis patients

The combination of two drugs — an investigational drug used in conjunction with an already FDA-approved medication — improved lung function in patients with one form of cystic fibrosis, according to two new studies. [More]
Combined therapy shows promise in cystic fibrosis patients

Combined therapy shows promise in cystic fibrosis patients

Treatment with two medications that target the most common genetic cause of cystic fibrosis improves lung function and lowers the rate of pulmonary exacerbations, according to the results from a Phase III international clinical trial published online in the New England Journal of Medicine on May 17, 2015. [More]
Gene therapy is key to addressing Sanfilippo Syndrome, say Ohio scientists and clinicians

Gene therapy is key to addressing Sanfilippo Syndrome, say Ohio scientists and clinicians

Gene therapy is the delivery of DNA into a patient's cells to replace faulty or missing genes—or adds new genes—in an attempt to cure cancer or make changes so the body is better able to fight off disease. Scientists and clinicians have identified a number of different ways to do this, in an effort to correct malfunctioning or mutated genes. Many gene therapy clinical trials are ongoing to assess the safety and potential benefits in patients with rare diseases. [More]
Penn study highlights need for more guidance on contraceptive use in women with cystic fibrosis

Penn study highlights need for more guidance on contraceptive use in women with cystic fibrosis

Only half of women with cystic fibrosis (CF) report using contraception and frequently apt to become pregnant unintentionally, according to a new study from researchers at the Perelman School of Medicine at the University of Pennsylvania. [More]
Adelaide researchers identify target for developing new antibiotics against disease-causing bacteria

Adelaide researchers identify target for developing new antibiotics against disease-causing bacteria

University of Adelaide researchers have discovered a target for the development of completely new antibiotics against disease-causing bacteria. [More]
Tuberous Sclerosis Alliance to observe fourth annual TSC Global Awareness Day

Tuberous Sclerosis Alliance to observe fourth annual TSC Global Awareness Day

On May 15, the Tuberous Sclerosis Alliance (TS Alliance) will join tuberous sclerosis complex (TSC) organizations around the world to observe the fourth annual TSC Global Awareness Day. [More]
Sangamo BioSciences to present data on ZFP Therapeutic platform at ASGCT meeting

Sangamo BioSciences to present data on ZFP Therapeutic platform at ASGCT meeting

Sangamo BioSciences, Inc. announced that data from clinical, preclinical and research-stage programs focused on the development of ZFP Therapeutics will be presented at the 18th Annual Meeting of the American Society of Gene and Cell Therapy [More]
Research findings pave way for development of new treatments for respiratory diseases

Research findings pave way for development of new treatments for respiratory diseases

The research has discovered a link between a vital pumping system that does not function correctly in people with cystic fibrosis and the parasite Toxoplasma. [More]
Yale researchers successfully correct gene mutation that causes cystic fibrosis

Yale researchers successfully correct gene mutation that causes cystic fibrosis

Yale researchers successfully corrected the most common mutation in the gene that causes cystic fibrosis, a lethal genetic disorder. The study was published April 27 in Nature Communications. [More]
New study sheds light on mechanism that affects AID enzyme

New study sheds light on mechanism that affects AID enzyme

A new study by immunology researchers at the IRCM led by Javier M. Di Noia, PhD, sheds light on a mechanism affecting AID, a crucial enzyme for the immune response. The scientific breakthrough, published in the latest issue of The Journal of Experimental Medicine, could eventually improve the way we treat the common flu, as well as lymphoma and leukemia. [More]
Humans carry recessive disease mutations that can cause severe genetic disorders or prenatal death

Humans carry recessive disease mutations that can cause severe genetic disorders or prenatal death

Humans carry an average of one to two mutations per person that can cause severe genetic disorders or prenatal death when two copies of the same mutation are inherited, according to estimates published today in the journal GENETICS. [More]
Study may point to immediate cause of CF exacerbations

Study may point to immediate cause of CF exacerbations

In the genetic disorder cystic fibrosis (CF), the most severe symptoms are recurring episodes of lung inflammation and bacterial infection (known as "exacerbations") that happen from one to three times a year and cause ever-increasing amounts of lung damage through the course of a CF patient's life. [More]
Research breakthrough could help develop tools to repair damaged nerve cells

Research breakthrough could help develop tools to repair damaged nerve cells

A team of researchers at the IRCM led by Frédéric Charron, PhD, in collaboration with bioengineers at McGill University, uncovered a new kind of synergy in the development of the nervous system, which explains an important mechanism required for neural circuits to form properly. [More]
New study opens door to preventative treatments for congenital diaphragmatic hernia

New study opens door to preventative treatments for congenital diaphragmatic hernia

Congenital diaphragmatic hernia (CDH) is not as well known as muscular dystrophy and cystic fibrosis, but like them it is a life-threatening birth defect, and is just as common. Occurring in one in 3,000 births, CDH causes the guts and liver to protrude through a defective diaphragm and into the chest cavity, where they interfere with the lungs. [More]
UCSF professor wins Vilcek Prize in Biomedical Science

UCSF professor wins Vilcek Prize in Biomedical Science

Peter Walter was chosen as winner of the Vilcek Prize in Biomedical Science for his pioneering work on how proteins are transported between cellular compartments and for unraveling the components of a regulatory mechanism that cells use to handle stress tied to the aggregation of misshapen proteins. [More]
Key finding may point to immediate cause of CF exacerbations

Key finding may point to immediate cause of CF exacerbations

In the genetic disorder cystic fibrosis (CF), the most severe symptoms are recurring episodes of lung inflammation and bacterial infection (known as "exacerbations") that happen from one to three times a year and cause ever-increasing amounts of lung damage through the course of a CF patient's life. [More]
Publicly insured Americans who undergo lung transplantation fare worse than their UK counterparts

Publicly insured Americans who undergo lung transplantation fare worse than their UK counterparts

Publicly insured Americans who undergo lung transplantation for cystic fibrosis fare markedly worse in the long run than both publicly insured patients in the United Kingdom and privately insured Americans, according to the results of a study conducted by researchers from Johns Hopkins in Baltimore and U.K. colleagues working in that nation's government-funded National Health Service. [More]
Male genetic diversity declined due to wealth, power rather than `survival of fittest`

Male genetic diversity declined due to wealth, power rather than `survival of fittest`

The DNA you inherit from your parents contributes to the physical make-up of your body -- whether you have blue eyes or brown, black hair or red, or are male or female. [More]
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