News

ELX-02 restored the levels and function of CFTR — the defective protein in CF — in “mini-organs,” or organoids, derived from patients carrying the most common CF-causing nonsense mutation, a study shows. “ELX-02 increased CFTR activity in a dose-dependent fashion,” with these functional increases being “similar to those obtained…

The National Institute for Health Research (NIHR) awarded a grant worth more than £1.5 million (about $2.1 million) to support a U.K. clinical trial investigating ways of safely lowering the treatment burden of cystic fibrosis (CF), a top priority for patients and their healthcare teams. The randomized and open-label…

Pharmaxis announced that a first batch of Bronchitol (mannitol), its dry powder and mucus clearing treatment for cystic fibrosis (CF), is on its way to the U.S. from the company’s manufacturing center in Australia. Recently approved by U.S. Food and Drug Administration (FDA) as a maintenance treatment for…

A recent survey shows that people with cystic fibrosis (CF) broadly favor targeted therapy using patient-derived organoids — the three-dimensional organ-like structures made from a patient’s own cells. The finding suggests that this technology might be integrated successfully into healthcare systems. The study, “Avatar acceptability: views…

Endoscopic sinus surgery led to better lung function in people with moderate or severe cystic fibrosis (CF) — and some with mild disease — with benefits sustained one year after surgery, a medical records review found. 

Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…

Blood C-reactive protein (CRP) and calprotectin levels can reliably identify pulmonary exacerbations in cystic fibrosis (CF) patients, making them promising diagnostic biomarkers, a pilot study has found. Although further investigations are necessary to determine their clinical applicability, the researchers note that “a step-wise algorithm that…

An annual retreat for mothers of children of all ages with cystic fibrosis (CF) — virtual again this year — will take place on Saturday, May 1, giving these moms a space in which to connect and refuel. Called “Embrace,” the retreat is hosted by Cystic Fibrosis Research…

Higher doses of vitamin D supplements do not appear to lead to better clinical outcomes, either in lung function or bone health, in people with cystic fibrosis (CF), a systematic review reported. Because this crucial vitamin is lacking in patients, however, further studies evaluating the long-term effects of vitamin…

While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs for prescription medicines and access to affordable comprehensive care — still need attention. Those were the findings of the…