Researchers and clinicians gather every year at the North American Cystic Fibrosis Conference to discuss the latest developments in cystic fibrosis research and care. During this one-hour webinar, James Lawlor, an adult with CF, hosts Raksha Jain, MD, and Jennifer Taylor-Cousar, MD, will share highlights from the conference and answer audience questions.
Among other research and care news, you will hear about the results of the Simplify study, which focused on whether it was safe for people with CF to stop inhaled hypertonic saline or dornase alfa (Pulmozyme®) while taking Trikafta®.
0:00 | NACFC highlights
02:21 │ Projected growth in the CF population over time
04:00 │ Higher lung function expected over time
05:51 │ Lung function and symptoms after Trikafta
07:35 │ Effect of withdrawal of medications after Trikafta (Simplify study)
10:58 │ Detecting infections post-Trikafta
14:06 │ NTM infections in CF
17:10 │ Some results from survey on well-being post-Trikafta
19:50 │ Liver disease in CF: new guidelines coming
22:09 │ Impact of Trikafta on the pancreas
25:02 │ Report on baby born to mother with CF on Trikafta
27:57 │ Use of Trikafta in people with CF post-lung transplant
36:22 │ Results of a study of Trikafta in children with CF ages 2-5
38:37 │ Lab test results of a potential new modulator
40:25 │ Discussion of genetic therapies in development
49:13 │ Sionna study: Is this a different formulation of Vertex’s 121 modulator study?
49:43 │ Do we know how close mRNA therapies are to phase 1,2,3 clinical trials?
50:10 │ Are people with CF able to take Paxlovid if they get COVID and they are also taking Trikafta?
51:36 │ How are we studying the effects of withdrawing medications while on Trikafta following the Simplify study?
52:46 │ Why do we use viruses to deliver genetic therapies?
53:36 │ Is CRISPR gene editing something that can be used in treating CF some day?
54:28 │ If a modulator study shows a decrease in sweat chloride, will that translate into a better clinical effect in patients?
56:00 │ Does Trikafta affect hormones in people with CF?
57:52 │ What research is happening to help people with nonsense mutations?
59:30 │ How do you try to balance the two patient populations — those with a modulator and those without — which are starting to have very different needs?
Originally recorded December 6, 2022.
