cystic fibrosis

While the majority of patients diagnosed with cystic fibrosis (CF) at birth experience the symptoms of the disease during their childhood, Larry Brian is an uncommon case. The 25-year old patient never really understood the extent of suffering from the chronic and debilitating disease until he went to college.

Proteostasis Therapeutics, Inc. (PTI), a biotechnology company working on breakthrough therapeutics to treat diseases caused by defects in protein processing, recently announced that it has secured new financing worth $37 million, which will be dedicated to further developing its primary product candidate for cystic fibrosis for testing in human clinical studies. Cystic fibrosis…

Basilea Pharmaceutica Ltd recently announced plans to expand the delivery system of the experimental antibiotic BAL30072 to include an inhaled form suitable for cystic fibrosis patients and those with bronchiectasis.  Both conditions are known for the presence of multi-drug resistant colonization of bacteria that can lead to respiratory…

Cystic Fibrosis researcher Dr. Joshua R. Stokell, PhD, 36, of Charlotte, NC, passed away on June 8, 2015, due to complications associated with his own lifelong battle with Cystic Fibrosis (CF). A graduate of the University of North Carolina at Charlotte, Dr. Stokell had studied Cystic Fibrosis after earning his…

A recent review entitled “Inhaled therapy in cystic fibrosis: agents, devices and regimens” published in the journal Breathe explored the increasing number of inhaled therapeutic options for patients with CF, and how innovations in these therapies are dramatically improving quality of life…

In a paper entitled “Targeted delivery of liquid micro volumes into the lung” published in the Proceedings of the National Academy of Sciences of the United States of America (PNAS), a multidisciplinary team of researchers from Columbia University in New…

Researchers at Institut national de la santé et de la recherche médicale (INSERM) in France have learned one way to “fix” cells that have characteristics of cystic fibrosis. These cells, which had faulty CFTR channel proteins, were able to make more functional CFTR protein following treatment with tumor necrosis…

Several studies have recently been released looking at the use of dry mannitol powder in an inhalation device for the management of cystic fibrosis. One study, entitled “Optimising inhaled mannitol for cystic fibrosis in an adult population,” published in Breathe, explored the safety and…

In both adolescents and adults, nutritional status can play a major role in the rate of disease progression. Individuals with poor nutritional status and low weight retention are at a higher risk for worsening lung function and increased mortality. However, in light of a new study from Hospital Universitario Ramón…

Future Science Group (FSG), a progressive publisher focused on breakthrough medical, biotechnological, and scientific research, has announced publication of a special issue in Future Science OA, covering the rapidly evolving field of protein misfolding diseases — a category that includes Cystic Fibrosis, Alzheimer’s and Parkinson’s…