@mondoterremoti Bravissimi! Magnifico thread, di grande chiarezza espositiva e capacità di spiegare fenomeni complessi senza banalizzare. Congratulazioni!
Real-World Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis: A 24-Month Italian National Registry Study https://t.co/D2sAR4MyV4 #mdpijcm via @JCM_MDPI
Nationwide longitudinal study using the Italian CF Registry (ICFR, ~97% national coverage).
A thread
Key limitations (stated in the paper)
• Annual best-FEV₁ only, cannot capture within-year variability
• No systematic PEx data
• No untreated control group
• Microbiological ascertainment bias (reduced sputum on ETI)
• No adherence data
Findings are hypothesis-generating.
Informazioni sul trattamento della Fibrosi cistica? https://t.co/O6UqE20MWP Un progetto di fondazione fibrosi cistica; oramai quando il sito viene aperto, il browser chiede se vuoi tradurre la pagina in italiano.
For over 11 years, CFDB – the Cystic Fibrosis DataBase – has provided up-to-date evidence on CF treatments and 50 continuously updated expert Topics. A key resource for anyone working in CF.
👉 https://t.co/O6UqE21kMn
Thanks to Fondazione Fibrosi Cistica for ongoing support.
#CF
Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Cystic Fibrosis: A Review of Registry-Based Evidence https://t.co/UiVbmDTu2u #mdpijcm via @JCM_MDPI
CFTR modulators changed the life of people with cystic fibrosis. Here is a review of this revolution.
This once-daily therapy showed better control of CF symptoms and could mean fewer hospital visits and more carefree childhoods. Starting treatment early might help prevent long-term complications. A big step forward for little warriors! 💪💙 #CysticFibrosis#StrongerTogether
Hopeful News for Kids with CF! 🌈
A recent study tested a new drug, Vanzacaftor-Tezacaftor-Deutivacaftor, in children aged 6–11 with CF. The results are promising: it’s safe, well-tolerated, and may improve lung health and quality of life even further than current treatments.
Vanzacaftor–tezacaftor–deutivacaftor versus elexacaftor–tezacaftor–ivacaftor in individuals with cystic fibrosis aged 12 years and older: results from two randomised, active-controlled, phase 3 trials - The Lancet Respiratory Medicine https://t.co/iIUejREc8w