News

Boston-based ProQr Therapeutics marked Rare Disease Day with an event commemorating one of its co-founders, rare disease pioneer Henri A. Termeer. The company develops treatments for such rare diseases as cystic fibrosis and dystrophic epidermolysis bullosa by creating transformative RNA medicines. Celebrated on the last day of February every year, Rare…

Cystic fibrosis patients in Canada may choose to use Flovent (fluticasone propionate), an inhaled corticosteroid that helps reduce inflammation in the lungs, to manage their symptoms. But the cost of such treatment often depends of where they live: In Nova Scotia, Flovent is covered by that provinces’s publicly…

The U.K.’s Medicines & Healthcare products Regulatory Agency (MHRA) issued an alert to urge patients and healthcare professionals to stop using Aquilon‘s medical nebulizers. Among those affected are patients with cystic fibrosis and asthma. All medical devices marketed in Europe need to have a mandatory CE certification,…

Vertex Pharmaceuticals is offering the England arm of Britain’s National Health Service a package deal on the pricing of its current and future cystic fibrosis therapies. What the company describes as “a bold new portfolio approach” to pricing comes in the wake of the National Health Service balking at covering…

Vertex Pharmaceuticals launched the first Phase 3 clinical trial investigating the triple combination therapy of VX-659, tezacaftor (VX-661), and Kalydeco (ivacaftor) in cystic fibrosis (CF) patients with one F508del mutation and one minimal function mutation in the CFTR gene, which is defective in CF patients. Vertex hopes the results…

A company that provides remote monitoring of patients’ health conditions has created patient and caregiver advisory boards for the lung conditions cystic fibrosis and pulmonary fibrosis. PMD Healthcare, which describes itself as a digital health and care management company, said CF and PF board members’ perspectives will help it do…

Santhera Pharmaceuticals has bought the rights to POL6014, a medication being developed by Polyphor to treat cystic fibrosis (CF) and other pulmonary diseases. Under terms of the global licensing agreement between the two Swiss companies, Santhera will develop, handle regulatory filings and market the investigative drug and derivate…

A Phase 1 clinical trial testing Arch Biopartners’ AB569 for the treatment of antibiotic-resistant bacterial infections is now enrolling healthy volunteers. The therapy candidate could be a new help for patients with cystic fibrosis (CF), chronic obstructive pulmonary disease (COPD), and other respiratory conditions. The trial will evaluate the…

AzurRx BioPharma announced that its clinical trial testing MS1819-SD as a replacement enzyme therapy for exocrine pancreatic insufficiency (EPI) associated with chronic pancreatitis (inflammation of the pancreas) has enrolled three new patients, completing 75 percent of its goal. Although patients with cystic fibrosis (CF) are excluded from the trial,…

An Australian scientist has received a $1 million Cystic Fibrosis Foundation grant to study whether probiotics can improve the health and life expectancy of children with the disease. Keith Ooi, a University of New South Wales pediatric gastroenterologist, will look at whether daily probiotics can improve the gut bacteria mix and…