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.
Bayer's Ciprofloxacin DPI receives FDA orphan drug designation for NCFB treatment

Bayer's Ciprofloxacin DPI receives FDA orphan drug designation for NCFB treatment

Bayer HealthCare today announced that the U.S. Food and Drug Administration's Office of Orphan Products Development has granted orphan drug designation for its investigational Ciprofloxacin Dry Powder for Inhalation (Ciprofloxacin DPI) for the treatment of non-cystic fibrosis bronchiectasis (NCFB). [More]
Researcher explores public perceptions related to newborn screening programs

Researcher explores public perceptions related to newborn screening programs

While 94 per cent of Canadians surveyed said they would participate in public health programs that screen newborns for a specific number of genetic conditions, only 80 per cent said they would be willing to participate in screening that would sequence their newborns' genomes. [More]
FTC requests additional information relating to Actavis’ pending acquisition of Forest Laboratories

FTC requests additional information relating to Actavis’ pending acquisition of Forest Laboratories

Actavis plc and Forest Laboratories, Inc. today announced that they have each received a request for additional information from the Federal Trade Commission ("FTC") in connection with Actavis' pending acquisition of Forest. [More]

OPKO Health enters into definitive agreement to acquire Inspiro Medical

OPKO Health, Inc. has entered into a definitive agreement to acquire Inspiro Medical Ltd., an Israeli medical device company developing a new platform to deliver small molecule drugs such as corticosteroids and beta agonists or larger molecules to treat respiratory diseases. Inspiro's Inspiromatic is a "smart" easy-to-use dry powder inhaler with several advantages over existing devices. [More]
Alcresta signs agreement with CFFT to support nutritional status of people with cystic fibrosis

Alcresta signs agreement with CFFT to support nutritional status of people with cystic fibrosis

Alcresta, a leading medical nutrition company developing innovative enzyme-based products for individuals with unique nutritional needs battling acute conditions or chronic diseases, today announced the company has signed an agreement with Cystic Fibrosis Foundation Therapeutics (CFFT) to accelerate the development of Alcresta's enzyme-based point-of-care products to support the nutritional status of people with cystic fibrosis (CF). [More]
FDA grants clearance for Breathe Technologies’ Non-Invasive Open Ventilation System

FDA grants clearance for Breathe Technologies’ Non-Invasive Open Ventilation System

Breathe Technologies, Inc. announced today that the U.S. Food and Drug Administration has granted the fifth 510(k) clearance for its Non-Invasive Open Ventilation System, allowing its use with compressed air supply for non-oxygen dependent patients. [More]

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

​A 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]