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.
Ironwood and Forest commence new DTC patient awareness campaign for LINZESS

Ironwood and Forest commence new DTC patient awareness campaign for LINZESS

awareness campaign for LINZESS, a once-daily treatment for adults with irritable bowel syndrome with constipation or chronic idiopathic constipation. [More]
Doc receives grant for helping young adults to move from child-centered to adult-oriented health care system

Doc receives grant for helping young adults to move from child-centered to adult-oriented health care system

Oscar Taube, M.D., director of the Pediatric Outpatient department and the coordinator of Adolescent Medicine at the Herman & Walter Samuleson Children's Hospital at Sinai, has been awarded a grant from the American Academy of Pediatrics to provide adolescents, young adults and their family members with the tools to make a smooth transition from pediatric medical care to adult medical care. [More]
Forest Laboratories, Almirall receive feedback from FDA regarding fixed dose combination of aclidinium/formoterol

Forest Laboratories, Almirall receive feedback from FDA regarding fixed dose combination of aclidinium/formoterol

Forest Laboratories, Inc. and Almirall, S.A. today announced that they have recently received feedback from the U.S. Food and Drug Administration (FDA) regarding the fixed dose combination of aclidinium and formoterol. [More]
Study shows critical role of immune system in protecting against viruses and cancer cells

Study shows critical role of immune system in protecting against viruses and cancer cells

‚ÄčA team of researchers at the IRCM, led by Andr- Veillette, MD, explains how our immune system kills abnormal blood cells. Their discovery, recently published in the Journal of Experimental Medicine, could eventually lead to new treatment avenues for leukemia, lymphoma and certain types of infectious viral diseases. [More]
Thomson Reuters, Children's Tumor Foundation partner to create neurofibromatosis pathway maps

Thomson Reuters, Children's Tumor Foundation partner to create neurofibromatosis pathway maps

The Intellectual Property & Science business of Thomson Reuters, the world's leading provider of intelligent information for businesses and professionals, today announced an expansion of its partnership with the Children's Tumor Foundation to create unique neurofibromatosis (NF) pathway maps aimed at significantly increasing the understanding of the disease and its underlying mechanisms. [More]
Forest Laboratories, Richter report positive results from cariprazine Phase IIb trial for bipolar depression

Forest Laboratories, Richter report positive results from cariprazine Phase IIb trial for bipolar depression

Forest Laboratories, Inc. and Gedeon Richter Plc. today announced positive topline results from a Phase IIb trial evaluating the efficacy and safety of the investigational antipsychotic cariprazine in patients with bipolar depression. [More]
Research findings help explain rare genetic disorder that causes immunodeficiency syndrome

Research findings help explain rare genetic disorder that causes immunodeficiency syndrome

IRCM researchers led by Javier M. Di Noia, PhD, uncovered a new function of AID, a crucial enzyme for the immune response. The discovery, recently published by the scientific journal Proceedings of the National Academy of Sciences, helps explain a rare genetic disorder that causes an immunodeficiency syndrome. [More]
Johns Hopkins researchers identify protein that regulates the body's immune response to CMV

Johns Hopkins researchers identify protein that regulates the body's immune response to CMV

Infectious disease specialists at the Johns Hopkins Children's Center have identified a protein that regulates the body's immune response to cytomegalovirus (CMV), a common pathogen that causes lifelong infections and can lead to devastating illness in newborns and those with weakened immune systems. [More]
Insmed reports results from ARIKAYCE phase 2 trial for treatment resistant NTM lung infections

Insmed reports results from ARIKAYCE phase 2 trial for treatment resistant NTM lung infections

Insmed Incorporated today reported results from the Company's phase 2 clinical trial of ARIKAYCETM, or liposomal amikacin for inhalation, for the treatment of patients with treatment resistant nontuberculous mycobacterial (NTM) lung infections. [More]
ASU scientist selected as 2014 recipient of Lifetime Achievement Award

ASU scientist selected as 2014 recipient of Lifetime Achievement Award

Roy Curtiss III, a scientist at the Biodesign Institute at Arizona State University, has been selected as the 2014 recipient of the Lifetime Achievement Award from the American Society for Microbiology. [More]
Aradigm reports record revenue of $4.6M in fourth quarter 2013

Aradigm reports record revenue of $4.6M in fourth quarter 2013

Aradigm Corporation (the "Company") today announced financial results for the fourth quarter and full year ended December 31, 2013. [More]

N30 Pharma begins oral dosing in N91115 Phase 1 trial for treatment of cystic fibrosis

N30 Pharmaceuticals, Inc. announced today that it has begun oral dosing of N91115 in a Phase 1 clinical trial. N91115, a novel inhibitor of S-nitrosoglutathione reductase (GSNOR), is being developed for the treatment of cystic fibrosis (CF). [More]

Genetic basis of hereditary disease causes severe brain atrophy in Jews of Moroccan ancestry

Ben-Gurion University of the Negev (BGU) researchers have unraveled the genetic basis of a hereditary disease that causes severe brain atrophy, mental retardation and epilepsy in Jews of Moroccan ancestry, according to a study published this week online in the Journal of Medical Genetics. [More]
Safeguard Scientifics reports net income of $24 million for fourth quarter 2013

Safeguard Scientifics reports net income of $24 million for fourth quarter 2013

Safeguard Scientifics, Inc. today announced fourth quarter and full-year 2013 financial results. For the three months ended December 31, 2013, Safeguard's net income was $24.0 million, or $1.10 per share, compared to net loss of $10.8 million, or $0.51 per share for the same period in 2012. For the year ended December 31, 2013, Safeguard's net loss was $35.5 million, or $1.66 per share, compared to $39.4 million, or $1.88 per share for the same period in 2012. [More]
Treating cystic fibrosis: an interview with Simon Bedson, Senior Vice President and International General Manager at Vertex

Treating cystic fibrosis: an interview with Simon Bedson, Senior Vice President and International General Manager at Vertex

Cystic fibrosis is a rare, life-shortening genetic disease for which there is no cure. It affects more than 75,000 people worldwide. In CF, thick, sticky mucus blocks the passages in many organs, leading to a variety of symptoms. In particular, mucus builds up and clogs airways in the lungs making it easier for bacteria to grow. [More]

Computational Fluid Dynamics helps patients with pulmonary disorders breathe easier

A more accurate and successful, yet complex approach used in designing an airplane is now taking off in the health care industry. [More]
Scientists find certain species of nontuberculous mycobacteria in water that cause infection in humans

Scientists find certain species of nontuberculous mycobacteria in water that cause infection in humans

Brisbane's water supply has been found to contain disease carrying bugs which can be directly linked to infections in some patients, according to a new study by QUT. [More]
Heat shock protein may be involved in folding processes of tau protein

Heat shock protein may be involved in folding processes of tau protein

Proteins like the so-called heat shock protein Hsp90 play an important role in almost all processes within human cells. They help other proteins fold into their three-dimensional structure or return damaged proteins back into their proper shape. [More]
Link found between pollutants and certain metabolic complications of obesity

Link found between pollutants and certain metabolic complications of obesity

A team of researchers at the IRCM in Montr-al led by R-mi Rabasa-Lhoret, in collaboration with J-r-me Ruzzin from the University of Bergen in Norway, found a link between a type of pollutants and certain metabolic complications of obesity. Their breakthrough, published online this week by the Journal of Clinical Endocrinology & Metabolism, could eventually help improve the prevention, diagnosis, and treatment of cardiometabolic risk associated with obesity, such as diabetes, hypertension and cardiovascular disease. [More]
FDA approves KALYDECO for people with cystic fibrosis

FDA approves KALYDECO for people with cystic fibrosis

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) approved a supplemental New Drug Application (sNDA) for KALYDECOTM (ivacaftor) for people with cystic fibrosis (CF) ages 6 and older who have one of eight additional mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. KALYDECO was first approved in January 2012 for people with CF ages 6 and older who have at least one copy of the G551D mutation. [More]