Combining investigational therapy MS1819 with standard pancreatic enzyme replacement therapy (PERT) can improve fat absorption in people with cystic fibrosis (CF) who have severe exocrine pancreatic insufficiency (EPI), top-line results from a Phase 2 clinical trial show. “A safe and effective therapy that allows CF patients to gain control over…
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Nathan Perdue, the father of a 2-year-old boy with cystic fibrosis (CF), in collaboration with the Kentucky Derby Museum, is raffling off his limited-edition collection of bourbons to support the Cystic Fibrosis Foundation (CFF) and its efforts to cure this disease within his son’s lifetime. The collection includes…
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…
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