Michael Boyle, MD, has laid out a five-year strategic plan for the Cystic Fibrosis Foundation (CFF), following his Jan. 1 appointment as president and CEO of the Maryland-based charity. In a three-minute video message posted Jan. 9 to the CFF website, Boyle said the 2020-24 plan will focus…
Trikafta, a recently approved treatment for cystic fibrosis (CF), is now available through the U.S. home delivery and specialty pharmacy AllianceRx Walgreens Prime, as part of its limited distribution therapeutic offerings. Trikafta is a next-generation combination of three CF medications: elexacaftor, tezacaftor, and ivacaftor.
Biopharmaceutical company Antabio has been awarded $4.4 million by CARB-X to continue its research for a therapy against Pseudomonas aeruginosa lung infections in people with cystic fibrosis (CF). The funding will be used to support Antabio’s Pseudomonas Elastase Inhibitor (PEi) program, which is seeking to develop…
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…
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…
Patients with cystic fibrosis (CF) have a high prevalence of chronic kidney disease (CKD) linked to higher levels of uric acid and fats in the blood, a study has found. These manifestations seem to be particularly severe in patients who receive a lung transplant. As such, performing early…
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…
It is well documented that patients with cystic fibrosis (CF) are a vulnerable population as far as nutrition is concerned. Daily energy needs for CF patients are typically one to one and a half…
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…
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