News

Vertex Pharmaceuticals is launching a new Phase 3 trial to assess the triple combination of VX-659 plus tezacaftor (VX-661) and Kalydeco (ivacaftor) in cystic fibrosis (CF) patients with two copies of the F508del mutation in the CFTR gene. That mutation most common CF-causing genetic variant. The trial will enroll about 100…

Children with cystic fibrosis who are able to recover from below-normal growth in their first two years are more likely to achieve normal growth and better lung condition by 12 years of age, a study shows. The research, “Early life growth patterns persist for 12 years and…

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…