cystic fibrosis

FDA Approves Trikafta to Treat Children Starting at Age 6

The U.S. Food and Drug Administration (FDA) has approved expanding the use of Trikafta (elexacaftor, tezacaftor, and ivacaftor) to children with cystic fibrosis (CF), ages 6 and older, who have at least one F508del mutation in the CFTR gene or a CFTR mutation that responds to Trikafta in laboratory studies. Use of…

New Cell Research Model Shows Promise Against P. Aeruginosa

A combination of bacteria-infecting viruses and antibiotics can be used to effectively protect lung cells against infection by Pseudomonas aeruginosa, a bacteria that commonly causes lung infections in people with cystic fibrosis (CF), according to a study led by researchers at the University of Geneva (UNIGE). The model…

CF Foundation Funds Three New Research Projects

As part of its Path to a Cure initiative, the Cystic Fibrosis Foundation announced three new awards to fund research projects that seek to advance the treatment of cystic fibrosis (CF). The largest award, of up to $2.6 million, was given to Eloxx Pharmaceuticals to aid in the…

Phage Therapy: Legacy of CF Advocate Mallory Smith Endures

The legacy of Mallory Smith, who died nearly four years ago from a cystic fibrosis-related superbug infection in her lungs, is living on in the form of raising awareness and funds for a novel treatment called phage therapy that might have saved her life.  Bacteriophages, or simply phages,…

Sildenafil Increases Exercise Capacity, Study Suggests

Sildenafil improves how muscles work when patients with cystic fibrosis (CF) exercise and this translates into better exercise capacity, a new study suggests. The study, “Exercise intolerance in cystic fibrosis: importance of skeletal muscle,” was published in the journal Medicine & Science in Sports &…

Tobramycin Given in Nanoparticles May Best Treat P. aeruginosa

A new biomaterial made up of nanoparticles showed a potential to improve — by up to 100,000 times — the efficiency of tobramycin, an antibiotic used to treat cystic fibrosis (CF) patients with Pseudomonas aeruginosa infections. Tobramycin, carried within a nanostructured material, completely eradicated P. aeruginosa infection in a…

Orkambi Shows Long-term Safety, Efficacy in Children Ages 2-5

Long-term use of Orkambi (lumacaftor/ivacaftor), an approved cystic fibrosis (CF) treatment, is safe and effective for children between ages 2 and 5 with two copies of the disease-causing F508del mutation in the CFTR gene, a study reported. It draws on results from a Phase 3 open-label extension trial…


Featured Column

How I Discovered That PTSD Was Causing My Post-transplant Struggles

PTSD, mental health, communities, stress, grateful, work, trauma, rare disease day, transplant, CF community, National Blood Donor Month, birthdays, holidays
Columnist Lara Govendo addresses an issue that several readers may relate to: post-transplant PTSD, which can wreak havoc.

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