cystic fibrosis

Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as scheduled, at the end of this month. Collin is an expert on the subject. For her University of Bath dissertation, she analyzed Brexit’s long-term impact…

Blocking the enzyme cathepsin S can alleviate symptoms and reduce lung damage in cystic fibrosis (CF) and chronic obstructive pulmonary disease (COPD), according to multinational teams led by researchers at Queen’s University Belfast, U.K. The findings on CF were reported in the study “Targeting of Cathepsin…

Like scores of runners everywhere, when Joshua Skampo gets home from work, he pulls on his running shoes and heads out into the dusk. But unlike most runners, Joshua runs for his life. He has cystic fibrosis (CF), a strength-sapping disease that makes it hard to breathe. Thick mucus…

ContraFect will receive up to $6.94 million in funding from CARB-X to support the development of their proprietary therapeutic peptides — amurins — against antibiotic-resistant bacterial infections caused by gram-negative ESKAPE pathogens. The company intends to develop these compounds as potential therapies for pulmonary exacerbations of cystic fibrosis and hospital-acquired…

With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases and the expensive therapies needed to treat them — particularly in an age of scarce economic resources — almost always entail “tragic choices,” warned Avraham Steinberg,…

Treatment of Pseudomonas aeruginosa lung infections in cystic fibrosis (CF) patients could be more effective if done within a crucial two- to three-year period when the bacteria are still susceptible to antibiotics, according to a study. The study, “Evolutionary highways to persistent bacterial infection,” was published in…

Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the estimated 7,000 rare diseases known to science have cures or treatments approved by the U.S. Food and Drug Administration (FDA). Raising awareness of those illnesses and highlighting…

An ongoing Phase 1/2 clinical trial exploring Translate Bio’s investigational therapy MRT5005 will start testing multiple ascending doses (MAD) in patients with cystic fibrosis (CF). This follows an assessment by a protocol review safety committee, which reviewed the study’s protocol and interim safety data. The committee approved…

MaRS Innovation recently announced the launch of two new projects targeting the development of new therapeutics for cystic fibrosis (CF) and respiratory syncytial virus (RSV). These projects are part of LAB150, MaRS Innovation’s joint venture with Evotec AG to support collaboration among scientists, investors, and pharmaceutical companies to…

Eloxx Pharmaceuticals will explore the potential of its lead investigational drug candidate, ELX-02, as a treatment for patients with cystic fibrosis (CF) caused by rare nonsense genetic mutations, in new trials integrated in the European HIT-CF project. “We are extremely excited to be participating in the HIT-CF research…