News

Register Now for Global Genes’ RARE Patient Advocacy Summit

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…

Lung Transplant Biorepository, Registry Targets Patient Outcomes

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…

Prime Editing, Advance on CRISPR, Shows Potential on CFTR Mutations

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…

Claire’s Place Foundation Glow Ride is Aug. 21

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…

NORD Rare Disease Summit, Online Oct. 18-19, Open for Registration

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…

Student Appointed to Ohio Rare Disease Advisory Council

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…