News

Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. Disorder: The Rare Disease Film Festival strives to eventually host a film about every one of the nearly 7,000 rare…

Proteostasis Therapeutics (PTI) has completed patient enrollment in a Phase 2 clinical trial evaluating its cystic fibrosis transmembrane conductance regulator (CFTR) modulator combination therapies in cystic fibrosis (CF) patients who have at least one copy of the F508del mutation in the CFTR gene (the defective gene in CF…

Latinos in the Caribbean with cystic fibrosis (CF) carry rare CFTR gene mutations not often seen in Latin or other CF populations elsewhere, a study led by physician-scientists at the University of California, San Francisco (UCSF) found. CF patients in Puerto Rico and the Dominican Republic have CFTR…

The bionic pancreas (BP), an automated system that controls blood sugar levels, may be useful in managing cystic fibrosis-related diabetes, a small study says. The study, “Automated glycemic control with the bionic pancreas in cystic fibrosis-related diabetes: A pilot study,” was published in the Journal of…

Eloxx Pharmaceuticals presented additional positive preclinical and clinical data for ELX-02, its lead treatment candidate for cystic fibrosis (CF), reinforcing the therapy’s safety and tolerability in humans and its ability to increase the levels of CFTR protein in preclinical models of the disease. Findings were presented in three posters and…

Preclinical data shows that Arrowhead Pharmaceuticals‘ investigational inhaled treatment for cystic fibrosis (CF), called ARO-ENaC, accelerates mucus clearance and preserves lung function while being safe for the kidneys in animal models. The data was shared recently at the 2019 North American Cystic Fibrosis Conference (NACFC) in Nashville,…

Two new alterations in the CFTR gene, leading to a deficient CFTR protein and consequently causing cystic fibrosis (CF), were identified in two young patients with the disease, according to a study. The study, “Two novel and correlated CF-causing insertions in the (TG)mTn tract of the CFTR gene,”…

Arrevus received a Small Business Research Loan from the North Carolina Biotechnology Center to support the development of the company’s Phase 3 therapeutic candidate ARV-1801 for the treatment of pulmonary exacerbations in patients with cystic fibrosis (CF). The Small Business Research Loan program grants investments of $75,000—$250,000…

The European Medicines Agency (EMA) has validated Vertex Pharmaceuticals’ application seeking approval for its triple combination — elexacaftor (VX-445), plus tezacaftor, and ivacaftor (Kalydeco) — for cystic fibrosis (CF) patients who cannot use other approved CF disease-modifying treatments or don’t benefit from them as intended.

The University of Queensland (UQ), in Australia, has launched its Queensland Cystic Fibrosis Research Program with a goal of improving the life of people with this disease. This research program is supported by $15 million donation put together by several sources: the Cystic Fibrosis Foundation in the U.S., and in Australia, UQ…