News

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,…

Vertex Pharmaceuticals is planning to request regulatory approval from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) during the third and fourth quarters of 2019, respectively, for VX-659 or VX-445 triple combination regimens. If approved, these new combo therapies will represent an alternative…

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…

NetNoggin has launched a new cystic fibrosis (CF) market research report to highlight patient and caregiver experiences and their perspectives about the gaps in CF research, the company announced. NetNoggin is a company that specializes in pharmaceutical marketing. The new offering, called NetNog: #genesis Cystic Fibrosis Patient and Caregiver…

Despite being commonly found in patients with cystic fibrosis-related diabetes, the bacteria Stenotrophomonas maltophilia cannot be considered as an independent risk factor for developing CFRD, study says. The study, “Cystic fibrosis related diabetes is not independently associated with increased Stenotrophomonas maltophilia infection: Longitudinal data from the UK CF Registry,”…

Exposing cystic fibrosis (CF) patients to high levels of oxygen through hyperbaric oxygen treatment may make Pseudomonas aeruginosa bacterial aggregates more sensitive to antibiotics like tobramycin and easier to eradicate from the lungs, a study in patient-derived bacterial cultures reports. The study, “Hyperbaric oxygen treatment increases killing of aggregating Pseudomonas aeruginosa isolates from…

Results from a Phase 3 trial demonstrate that Symkevi (tezacaftor and ivacaftor combo), known as Symdeko in the U.S., is safe and can effectively improve lung clearance in children, from 6 to 11 years old, with cystic fibrosis (CF). With these results, Vertex Pharmaceuticals — the…

Later this month, the Cystic Fibrosis Foundation (CFF) will host ResearchCon, its first virtual event dedicated to cystic fibrosis (CF) science and research. Some 480 people have registered for the Feb. 28 online conference, with at least 300 of them likely to participate, said Shellie Byrum, director of community…