An early clinical trial testing inhaled cystic fibrosis (CF) treatment VX-522 — being developed for adults with CF who are not eligible for certain available therapies — has been terminated before its completion due to “persistent tolerability issues,” according to an update from Vertex Pharmaceuticals. Vertex, which was…
Blackstone Life Sciences is investing $250 million in Anagram Therapeutics to advance ANG003, an oral enzyme replacement therapy (ERT) for exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF). ANG003 is designed to reduce the “enormous, disruptive” pill burden for CF patients with EPI, which can amount…
Discussion
Last in a series. Read part one. Last week, I asked why those with cystic fibrosis (CF), a progressive and incurable genetic disease, must keep proving disability after already qualifying. This is especially frustrating after reaching an advanced disease stage and undergoing a life-extending double-lung transplant, as I…
Discussion
Cystic fibrosis (CF) tends to take up a lot of space in my life. Sometimes it’s so all-consuming that I don’t get to focus on anything else, and I must wrestle to separate my identity from this label that I never chose. But CF is not my whole life…
Having a family history of diabetes may significantly increase the risk of developing cystic fibrosis-related diabetes (CFRD), a common complication of cystic fibrosis (CF), according to a new U.S. study. The researchers found that adults with CF who had a family history of diabetes, particularly type 2 diabetes,…
Discussion
My column, “Mutations & Conversations,” was first launched in 2018 after I observed Cystic Fibrosis Awareness Month by writing a blog post every day in May about living with cystic fibrosis (CF). This decision to write this series was made in the midst of grief: My sister,…
Children with cystic fibrosis who carry two Trikafta-responsive mutations are significantly more likely to reach normal or near-normal sweat chloride levels, suggesting stronger restoration of CFTR protein function than children with one responsive and one nonresponsive mutation, according to a real-world study. While this biochemical benefit ultimately did…
Perspectives
Newly diagnosed? Start your journey here.
Need to know
Becoming educated and informed about cystic fibrosis is a good first step. Here you’ll find general information about the disease, including diagnosis, symptoms, and causes.
Personal experiences
It helps to know others have been where you are now. Here’s a collection of our columnists’ words of wisdom to help you along on your CF journey.
Treatment
There is no cure for CF yet, but here you can find more information about current therapeutic approaches, as well as experimental treatments that are in the pipeline.