In June 2025, Dr. Julia Matilainen, representing the PCH2cure-project, attended the “Rare as One” meeting of the Chan Zuckerberg Initiative in Nevada, USA.
“Rare as One” – promoting patient-led organizations for rare diseases
The “Rare as One” network supports patient-led organizations in accelerating research of rare diseases, networking and promoting collaboration between researchers and doctors.
Annual exchange meeting between the organizations
Every year, the members of the network meet in person to listen to exciting presentations, find out about the latest activities of others and share their own progress.
There were exciting presentations with one coming from Fyodor Urnov, Professor at UC Berkeley and the Innovative Genomics Institute, who was involved in the development of the first CRISPR gene therapy for “Baby KJ” (see also our “Meet the Expert” webinar on this topic).
Furthermore, a series of interactive workshops on topics such as “Infrastructure and Tools for Cross-Disease Research” and “Unlocking Syngergies in Disease Manifestations” were offered. To the organizations, this provided the opportunity for exchange across different diseases and to explore ways of collaboration in order to make the best possible use of synergies and resources.
In between, there was enough time for personal exchange to learn from each other. As always, it was an enriching experience from which many ideas and learnings for PCH2cure were taken away.
