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.

NC Senate plan to overhaul state Medicaid program draws fire from doctors and hospitals

The North Carolina plan was presented by state senators during a Wednesday meeting of the Senate Rules and Operations Committee. [More]
First Edition: July 17, 2014

First Edition: July 17, 2014

Today's headlines include reports about how Capitol Hill politics are impacting health care policy. Houston Public Media's Carrie Feibel, working in partnership with Kaiser Health News and NPR, reports: "Primary care doctors have reported problems making referrals for patients who have purchased some of the cheaper plans from the federal insurance marketplace. [More]
PTC Therapeutics initiates reimbursed expanded access program for Translarna

PTC Therapeutics initiates reimbursed expanded access program for Translarna

PTC Therapeutics, Inc. today announced the initiation of a reimbursed expanded access program (EAP). PTC's EAP program is intended to make Translarna (ataluren) available to patients before commercial availability in certain countries. [More]
Children who undergo simple emergency surgeries on weekends are more likely to die

Children who undergo simple emergency surgeries on weekends are more likely to die

Children who undergo simple emergency surgeries, such as hernia repairs or appendix removals, on weekends are more likely to suffer complications and even die than children getting the same kind of treatment during the week, according to results of a Johns Hopkins Children's Center study. [More]
Vertex receives positive opinion from European CHMP for approval of KALYDECO for cystic fibrosis

Vertex receives positive opinion from European CHMP for approval of KALYDECO for cystic fibrosis

Vertex Pharmaceuticals Incorporated today announced that the European Committee for Medicinal Products for Human Use has issued a positive opinion recommending the approval of KALYDECO (ivacaftor) for people with cystic fibrosis (CF) ages 6 and older who have one of eight non-G551D gating mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. [More]
Queen's doctor plays key role in major breakthrough to change lives of cystic fibrosis sufferers

Queen's doctor plays key role in major breakthrough to change lives of cystic fibrosis sufferers

A Queen's University doctor has played a key role in a major breakthrough to change the lives of cystic fibrosis sufferers. [More]
Forest Laboratories reports positive results from three Phase III trials of vilazodone for GAD treatment

Forest Laboratories reports positive results from three Phase III trials of vilazodone for GAD treatment

Forest Laboratories, Inc. today announced positive topline results from three Phase III trials evaluating the efficacy, safety and tolerability of vilazodone in adult patients with generalized anxiety disorder (GAD). [More]
Aradigm begins Pulmaquin Phase III study for treatment of non-CF BE

Aradigm begins Pulmaquin Phase III study for treatment of non-CF BE

Aradigm Corporation ("Aradigm" or the "Company") today announced the dosing of the first patient in the ORBIT-4 (Once-daily Respiratory Bronchiectasis Inhalation Treatment) Phase III pivotal clinical trial of Aradigm's proprietary formulation of inhaled ciprofloxacin (Pulmaquin®) for the treatment of non-cystic fibrosis bronchiectasis (non-CF BE). [More]
Celsus’ expert highlights serious side effects of topical corticosteroids

Celsus’ expert highlights serious side effects of topical corticosteroids

A 2013 Mayo Clinic study involving more than 140,000 patients revealed that skin disorders are the most common nonacute reason Americans seek health care. [More]
Aradigm’s common shares approved for listing on NASDAQ Capital Market

Aradigm’s common shares approved for listing on NASDAQ Capital Market

Aradigm Corporation is pleased to announce that its common shares have been approved for listing on the NASDAQ Capital Market. [More]
Molecular mechanism linked to increased risk of diabetes in people with cystic fibrosis

Molecular mechanism linked to increased risk of diabetes in people with cystic fibrosis

Many people with cystic fibrosis develop diabetes. The reasons for this
have been largely unknown, but now researchers at Lund University in Sweden and
 Karolinska institutet have identified a molecular mechanism that contributes to
the raised diabetes risk. [More]
Forest publishes data from pivotal Phase III Study of Investigational FDC of nebivolol and valsartan

Forest publishes data from pivotal Phase III Study of Investigational FDC of nebivolol and valsartan

Forest Laboratories, Inc. (NYSE:FRX) today announced the publication of data from its pivotal Phase III Study of an Investigational Fixed-Dose Combination (FDC) of nebivolol and valsartan in the May 30, 2014 issue of The Lancet. [More]

Cystic fibrosis market estimated to reach $4.5 billion in 2019

Reportbuyer.com has added a new market research report: Cystic Fibrosis Therapeutics in Major Developed Markets to 2019 - CFTR Modulators Initiate Drive towards Personalized Treatment and Market Growth [More]
EMA's CHMP gives positive opinion for PTC Therapeutics' Translarna

EMA's CHMP gives positive opinion for PTC Therapeutics' Translarna

PTC Therapeutics, Inc. today announced that following its request for re-examination, the Committee for Medicinal Products for Human Use of the European Medicines Agency has adopted a positive opinion regarding the company's application for a conditional marketing authorization of Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged five years and older. [More]
Aradigm receives QIDP designation for its inhaled antibiotic candidate, Pulmaquin

Aradigm receives QIDP designation for its inhaled antibiotic candidate, Pulmaquin

Aradigm Corporation (the "Company") today announced that the U.S. Food and Drug Administration (FDA) has designated the Company's lead inhaled antibiotic candidate, Pulmaquin®, as a Qualified Infectious Disease Product (QIDP). [More]
Study shows technology improves transition into adulthood for adolescents with chronic disease

Study shows technology improves transition into adulthood for adolescents with chronic disease

Adolescents with chronic diseases (ACD), such as cystic fibrosis, gastrointestinal disorders (including Crohn's disease) and Type 1 diabetes, often find the transition of managing their health care needs into adulthood to be challenging. [More]
Aradigm reports total revenue of $6.6 million for Q1 2014

Aradigm reports total revenue of $6.6 million for Q1 2014

Aradigm Corporation today announced financial results for the first quarter and three months ended March 31, 2014. [More]
Trends, opportunities and threats in the life science industry: an interview with Bob Coughlin, CEO, MassBio

Trends, opportunities and threats in the life science industry: an interview with Bob Coughlin, CEO, MassBio

We were extremely excited to launch the Impact 2020 report at our annual meeting. Our five-year strategic plan is focused on value, early-stage financing and ensuring more patient-centered discussions. [More]
FDA approves Parion’s investigational new drug application for treatment of dry eye disease

FDA approves Parion’s investigational new drug application for treatment of dry eye disease

Parion Sciences, a company dedicated to the development of novel treatments for ocular and pulmonary diseases, announced today that it has received acceptance from the U.S. Food and Drug Administration (FDA) of its Investigational New Drug (IND) application for P-321 Ophthalmic Solution for the treatment of dry eye disease. [More]
Vertex: Conditions of PBAC recommendation may limit Australians who could benefit from KALYDECO

Vertex: Conditions of PBAC recommendation may limit Australians who could benefit from KALYDECO

The Pharmaceutical Benefits Advisory Committee has again recommended KALYDECO (ivacaftor) for listing on the Pharmaceutical Benefits Scheme (PBS). However, Vertex is deeply disappointed in the conditions the PBAC has attached to the recommendation. These conditions fail to reflect the substantial clinical benefits recognised by the PBAC, and would result in limiting the number of eligible Australians who would be able to receive KALYDECO. [More]