cystic fibrosis

People with cystic fibrosis (CF), like their healthy counterparts, often neglect sleep. But that can lead to problems down the road, warned three CF experts at the 2019 North American Cystic Fibrosis Conference in Nashville. More than half of all CF patients have sleep complaints, said pulmonologist Eliot…

Genetic variations that lower the expression levels of a gene called RNF5 may explain why some people with cystic fibrosis (CF) are at lesser risk of developing lung infections, a study reports. The study, “Systematic genetic analysis of the MHC region reveals mechanistic underpinnings of HLA type associations with…

Cystic Fibrosis News Today brought you daily coverage of important discoveries, treatment developments, clinical trials, and other important events related to cystic fibrosis (CF) throughout 2019. As we look forward to bringing you more CF news this year, we present our 10 most-read stories of 2019. No. 10 –…

Oregon retiree Carol Birch, 59, considers herself especially lucky to have made it this far in life. “I’ve never felt that I fit the typical role of someone with cystic fibrosis (CF),” Birch told attendees at the 2019 North American Cystic Fibrosis Conference (NACFC) in Nashville, Tennessee. Diagnosed at…

A triple combination of dirocaftor (PTI-808), Proteostasis Therapeutics’ investigational CFTR potentiator, with a proprietary CFTR amplifier and corrector led to significant improvements in lung function in patients with cystic fibrosis (CF) carrying the F508del mutation, according to topline data from a Phase 2…

Patients with cystic fibrosis (CF) in the Republic of Ireland will be among the first in Europe to benefit from Vertex Pharmaceuticals’ triple combination regimen composed of elexacaftor (VX-445), tezacaftor, and Kalydeco (ivacaftor), upon its approval by the European Medicines Agency. Following Vertex’s negotiations with the Republic of…

Babies diagnosed with cystic fibrosis (CF) through newborn bloodspot screening show better lung function, can better stave off infection with Pseudomonas aeruginosa (the most common bacteria found in CF), and have better weight outcomes than those diagnosed clinically later in life, a large CF registry-based study from the…

Three investigative therapies by Proteostasis Therapeutics — PTI-801, PTI-808, and PTI-428 — showed potential to treat cystic fibrosis (CF) patients ineligible for approved CFTR modulators after patient-specific lab models produced promising results that support a future clinical trial. Funded by the EU’s…

Preston W. Campbell III, MD, recently bid farewell as president and CEO of the Cystic Fibrosis Foundation (CFF) in Nashville — the same city where he began his long association with the disease 30 years ago as a pediatric pulmonologist at Vanderbilt University’s Cystic Fibrosis (CF) Care Center.

The European Commission has approved a label extension for Kalydeco (ivacaftor), allowing the therapy to be used in infants with cystic fibrosis (CF) who are between the ages of six months and one year. The decision follows a positive opinion on Kalydeco, marketed by Vertex Pharmaceuticals, that…