News

Verona Pharma’s nebulized RPL554 can significantly improve lung function in patients with cystic fibrosis, according to top-line data from a Phase 2a clinical trial. The latest results revealed that single administration of both high and low doses of the investigative drug significantly increased patients’ average forced expiratory volume in…

Caregivers of people with rare diseases face emotional, financial, physical, and social strain and receive little outside support, a U.S. study found. The findings will be presented to Congress this spring along with policy recommendations to help support those caring for people with rare diseases. The report is titled “…

Pulmonary hypertension increases the risk of cystic fibrosis patients’ lung function worsening, but is not a predictor of reduced survival, a study reports. The research, ”Prognostic significance of pulmonary hypertension in patients with cystic fibrosis: A systematic review and meta-analysis,” was published in the journal Medicine.

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…