We would like to express our sincere and heartfelt thanks to the n=1 Collaborative for their remarkable work over the past years. Without your commitment, many of the developments we are witnessing today would simply not have been possible. Thanks to your efforts in connecting people, sharing knowledge, and fostering true collaboration across borders, numerous new therapies are now on a promising path. What makes this community so unique is not only its scientific ambition, but also its courage. You bring together families, clinicians, and researchers to confront some of the most devastating conditions imaginable — and you do so with openness and integrity. This includes the willingness to share not only successes, but also setbacks and side effects. Only through this transparency can we truly learn from one another and move forward. We are convinced that this platform will remain of critical importance in the years ahead. Continued progress will depend on your courage to act, to collaborate, and to keep pushing boundaries — always with the shared goal of helping children affected by these rare and devastating diseases. It was a great honor for me to open the annual meeting in Budapest and to share the story of our daughter Valeria and our journey with all of you. Moments like these remind us how powerful this community truly is. Thank you for everything you do — your work gives hope where it is needed most.
