What have our PhD students been up to?
The 48th Europe Cystic Fibrosis Conference was held for 3 – 7 June 2025, in Milan, Italy. From the ORGESTRA community, two PhD students, Jorge Madrid Paredes (DC13) and Woosub Shin (DC12), and two PIs, Kors van der Ent and Jeffrey Beekman, attended this event. Jorge presented a poster on his plans to conduct a patient preference study with people and caregivers living with cystic fibrosis. Woosub distributed his questionnaire survey with the help of two ORGESTRA PIs, Kors van der Ent and Danya Muilwijk. During the conference, Kors and Jeffrey participated in presentations and discussions on several topics including therapeutic opportunities for people with cystic fibrosis with (ultra) rare variants and quality of life assessment via the Q-Life app. As PhD students, Jorge and Woosub attended several sessions to learn about recent developments and open challenges in cystic fibrosis treatments research, such as the current priorities of cystic fibrosis research. It was also a great opportunity to get connected with other researchers studying similar topics, such as those from COS-CF. During the conference, ORGESTRA researchers also had a friendly get-together.
By the way… what is cystic fibrosis?
Cystic fibrosis is a rare genetic disease caused by mutation of the cystic fibrosis transmembrane conductance regulator (CFTR) gene, causing lung inflammation, mucus buildup in the pancreas, and many other symptoms as a result. More than 162,000 individuals are affected worldwide. The treatment of cystic fibrosis went through a recent breakthrough with the development of CFTR modulators; for example, elexacaftor/tezacaftor/ivacaftor (ETI) has been found to significantly improve the symptoms of at least 85% of the patient population. On the other hand, the treatment options for those with rare variants and those not eligible for the current CFTR modulators are limited. Researchers are exploring new CFTR modulators that can help the remaining population as well as other treatment options such as gene therapy, where the research of ORGESTRA is highly relevant. Using emerging organoid models and other relevant methods, ORGESTRA researchers are hoping to contribute to this exciting and challenging field.
Thoughts of our DCs
“It was a great opportunity to learn about recent developments in cystic fibrosis research and connect with other researchers from all over Europe and beyond. Milan is a beautiful city with great options to go out and I was happy to have visited there for the conference. Looking forward to going back to next year’s conference to discuss more about the progress of my research.”
“It was a nice opportunity to share the study design for my qualitative patient preference study with the CF community. In the study, I will explore the preferences and opinions of people with cystic fibrosis and their caregivers. I also shared insights and contact details with researchers from the UK working on a similar study.”
