News

Patients with cystic fibrosis (CF) have a high prevalence of chronic kidney disease (CKD) linked to higher levels of uric acid and fats in the blood, a study has found. These manifestations seem to be particularly severe in patients who receive a lung transplant. As such, performing early…

A triple combination of dirocaftor (PTI-808), Proteostasis Therapeutics’ investigational CFTR potentiator, with a proprietary CFTR amplifier and corrector led to significant improvements in lung function in patients with cystic fibrosis (CF) carrying the F508del mutation, according to topline data from a Phase 2…

It is well documented that patients with cystic fibrosis (CF) are a vulnerable population as far as nutrition is concerned. Daily energy needs for CF patients are typically one to one and a half…

Three investigative therapies by Proteostasis Therapeutics — PTI-801, PTI-808, and PTI-428 — showed potential to treat cystic fibrosis (CF) patients ineligible for approved CFTR modulators after patient-specific lab models produced promising results that support a future clinical trial. Funded by the EU’s…

Preston W. Campbell III, MD, recently bid farewell as president and CEO of the Cystic Fibrosis Foundation (CFF) in Nashville — the same city where he began his long association with the disease 30 years ago as a pediatric pulmonologist at Vanderbilt University’s Cystic Fibrosis (CF) Care Center.

The European Commission has approved a label extension for Kalydeco (ivacaftor), allowing the therapy to be used in infants with cystic fibrosis (CF) who are between the ages of six months and one year. The decision follows a positive opinion on Kalydeco, marketed by Vertex Pharmaceuticals, that…

Treatment with Kalydeco (ivacaftor) alone improved lung function and quality of life in a cystic fibrosis patient carrying both a therapy-responsive and a non-responsive mutation to treatment, a case study reports. The case study, titled “Positive clinical outcomes following ivacaftor treatment in a cystic fibrosis patient with the…

The Government of Quebec, in Canada, has approved a loan of $2.7 million Canadian (about $2.05 million U.S.) to Laurent Pharmaceuticals to help the company conduct a Phase 2 clinical trial to evaluate its investigational oral candidate LAU-7b for…

Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.

Researchers in Israel and the U.S. have developed a new technology that can be used to unclog and eliminate mucus secretions from the airways of patients with various respiratory diseases, including cystic fibrosis (CF). The announcement was made by BGN Technologies, the technology company of Ben-Gurion University…