News

Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats also are available for attending the event in person in San Diego. “Here you’ll have the opportunity to connect and engage with others…

The Cystic Fibrosis Foundation (CFF) and the Cleveland Clinic are launching the Cystic Fibrosis Lung Transplant Consortium (CFLTC) Biorepository and Registry, aimed at supporting research into understanding the factors that influence lung transplant outcomes. “The biorepository and registry for lung transplant recipients represents an exciting leap forward in…

Food & Friends will be delivering medically tailored meals to people living with cystic fibrosis (CF) in the Washington, D.C., area, as part of a new collaboration with the Cystic Fibrosis Foundation. The partnership aims to serve CF patients who cannot shop for and prepare food for themselves.

Researchers corrected mutations underlying cystic fibrosis (CF) in a three-dimensional (3D) cell model of the disorder, using a new form of gene editing. This work serves as a proof-of-principle for the technique — called prime editing, seen as an improvement on the CRISPR/Cas9 gene editing tool — and raises the…

Researchers at the University of Massachusetts (UMass) Amherst have developed a new method of RNA production that leads to higher yields and purity of RNA molecules, likely making it more cost-effective than current approaches. Their work may be of importance to the production of RNA-based therapies for cystic fibrosis (CF)…

The seventh annual Glow Ride for Cystic Fibrosis, hosted by Claire’s Place Foundation, will resume along the coast of Los Angeles, California, on Aug. 21. Last year’s cystic fibrosis (CF) fundraising event was postponed out of consideration for safety measures related to the global COVID-19 pandemic. “Since…

Registration is now open for the 2021 Rare Diseases and Orphan Products Breakthrough Summit, which will be held virtually Oct. 18–19. The event, also known as the National Organization for Rare Disorders (NORD) Summit, brings the rare disease community together to network and discuss developments in treatments and research…

Andrea Hoffman, an Ohio Northern University (ONU) student who also is a cystic fibrosis (CF) patient and advocate, has been appointed by Ohio Gov. Mike DeWine to the Ohio Rare Disease Advisory Council, according to a university press release. In her two-year term, Hoffman, an ONU junior, will…

Poor bone density is increasingly evident in older cystic fibrosis (CF) patients, and may be linked to an inflammation biomarker known as interleukin 8 (IL-8), a small study in Canada suggests. Bone disorders can affect 24% of adults with CF, the study noted, adding that the Cystic Fibrosis Foundation…

Kaftrio successfully improved lung function and quality of life in three Italian cystic fibrosis (CF) patients who have severe lung disease with an unknown mutation in the CFTR gene in addition to the common F508del mutation, a case series reported. Kaftrio (elexacaftor, tezacaftor, and ivacaftor), sold as Trikafta in the…