Cystic Fibrosis News and Research

Latest Cystic Fibrosis News and Research

Pediatricians should screen all children for vitamin D deficiency

As study after study shows the fundamental role vitamin D plays in disease and health, vitamin D deficiency — which often develops insidiously in childhood — should be on every parent's and pediatrician's radar, say physicians from the Johns Hopkins Children's Center.

23 Feb 2012

Low choline intake increases risk of liver scarring in menopausal women with NAFLD

Menopausal women with non-alcoholic fatty liver disease (NAFLD) who don't consume enough of the essential nutrient choline appear to be at higher risk for liver scarring, according to research led by scientists at Johns Hopkins Children's Center.

17 Feb 2012

Cystic fibrosis drug gets FDA approval

A drug – the first of its kind - that treats the root cause of cystic fibrosis won approval Tuesday. This could be a ray of hope for patients with the deadly illness and broader hope for thousands more patients with the inherited disease.

1 Feb 2012

Vertex receives FDA approval for Kalydeco to treat rare form of CF

The U.S. Food and Drug Administration today approved Kalydeco (ivacaftor) for the treatment of a rare form of cystic fibrosis (CF) in patients ages 6 years and older who have the specific G551D mutation in the Cystic Fibrosis Transmembrane Regulator (CFTR) gene.

1 Feb 2012

LCI feasible, sensitive non-invasive marker of early lung disease in young children with CF

The lung clearance index (LCI) is a sensitive non-invasive marker of early lung disease in young children with cystic fibrosis (CF), according to a new study from Australian researchers.

27 Jan 2012

Platelet receptor could help with diseases that impact RBCs

A receptor found on blood platelets whose importance as a potential pharmaceutical target has long been questioned may in fact be fruitful in drug testing, according to new research from Michigan State University chemists.

20 Jan 2012

Pediatric research project to expand data collection linked to newborn screening results

Within the first days of life, screening tests are performed on all newborns born in the U.S. to identify rare and often life-threatening medical conditions that are not apparent at birth. These newborn screening programs have been operating for decades, permitting doctors to intervene early and improve outcomes in potentially devastating conditions.

18 Jan 2012

UI chemists debunk misconception about amphotericin

With one simple experiment, University of Illinois chemists have debunked a widely held misconception about an often-prescribed drug.

17 Jan 2012

COPDF announces expansion of Bronchiectasis Research Registry

The COPD Foundation (COPDF) today announced expansion of its Bronchiectasis Research Registry to include patients with Nontuberculous Mycobacteria (NTM), an often misdiagnosed orphan disease that can cause severe lung infections that mimic tuberculosis when certain individuals are exposed to soil and water.

12 Jan 2012

Studies identify genes and small molecules against protein conformational diseases

Two related studies from Northwestern University offer new strategies for tackling the challenges of preventing and treating diseases of protein folding, such as Alzheimer's, Parkinson's and Huntington's diseases, amyotrophic lateral sclerosis (ALS), cancer, cystic fibrosis and type 2 diabetes.

7 Jan 2012

Celtaxsys announces acquisition of CTX-4430 from Estrellita

Celtaxsys, Inc. a privately held biotechnology company, announced today that it has acquired CTX-4430 (formerly EP-501), a clinical stage drug candidate that inhibits the pro-inflammatory enzyme Leukotriene A4 Hydrolase (LTA4H), from Estrellita Pharmaceuticals, Inc.

6 Jan 2012

UCSF professor receives 2012 Paul Ehrlich and Ludwig Darmstaedter Prize

Peter Walter, PhD, a professor in the Biochemistry and Biophysics Department within the School of Medicine at the University of California, San Francisco has been awarded the 2012 Paul Ehrlich and Ludwig Darmstaedter Prize for his "outstanding research achievements in the field of cell biology."

26 Dec 2011

Researchers identify second oldest human disease mutation

A new study has identified a gene mutation that researchers estimate dates back to 11,600 B.C., making it the second oldest human disease mutation yet discovered.

16 Dec 2011

FDA accepts Vertex KALYDECO NDA for six-month Priority Review

Vertex Pharmaceuticals Incorporated announced today that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for KALYDECOTM (ivacaftor) and granted the company's request for six-month Priority Review.

16 Dec 2011

Gene therapy can reduce painful bleeding episodes in patients with hemophilia B

Symptoms improved significantly in adults with the bleeding disorder hemophilia B following a single treatment with gene therapy developed by researchers at St. Jude Children's Research Hospital in Memphis and demonstrated to be safe in a clinical trial conducted at the University College London (UCL) in the U.K.

12 Dec 2011

Israeli Nobel Laureate receives AvH's Humboldt Research Award

The Israeli Nobel Laureate Aaron Ciechanover has been named one of the recipients of the Humboldt Research Award of the Alexander von Humboldt Foundation.

8 Dec 2011

Positive results from Pearl Therapeutics' PT001 Phase 2b trial on COPD

Pearl Therapeutics Inc. today announced positive top-line results from a randomized, double-blind, Phase 2b, dose-ranging study of glycopyrrolate, a long-acting muscarinic agonist (LAMA) delivered twice a day (BID) via metered-dose inhaler in patients with moderate-to-severe COPD.

8 Dec 2011

New NHGRI funding plan sharpens focus of Genome Sequencing Program on medical apps

A new funding plan by the National Human Genome Research Institute (NHGRI) sharpens the focus of its flagship Genome Sequencing Program on medical applications. In addition to continuing on-going studies, the four-year, $416 million plan launches new efforts to find causes of rare inherited diseases and accelerate the use of genome sequence information in the medical care of patients.

7 Dec 2011

Towards a national plan of action for rare diseases

"Rare diseases", by their very definition, occur in no more than 5 people out of every 10,000 inhabitants. Barely noticed by the general public, only around 1,000 of the currently 6,000 or so different rare diseases currently listed on the Internet platform Orphanet are treatable nowadays.

5 Dec 2011

Doctors underestimate worldwide burden of CPA, says public health expert

Researchers have discovered that over a million people worldwide diagnosed with TB go on to develop an incurable but manageable fungal infection which is usually left untreated because it is mistaken for a recurrence of the disease.

2 Dec 2011