On 29 April 2025, the Institut de Ciència de Materials de Barcelona (ICMAB-CSIC) hosted Fabry Connections: Science, Patients, and Future, a special event designed to foster dialogue between researchers, clinicians, patients, and patient associations working to tackle Fabry disease. The event was framed within the European-funded Nano4Rare project, coordinated at ICMAB, which aims to develop an innovative nanomedicine to treat this rare lysosomal storage disorder.
The Nanomol-bio group at ICMAB-CSIC, deeply involved in the development of the Nano4Rare nanotherapeutic, played a central role in organizing and shaping the event. Several of the group’s researchers—who are at the forefront of nanomedicine and drug delivery research—helped prepare the programme, design the interactive sessions, and ensure that both scientific and patient voices were equally represented.
One of the highlights of the event was the opening presentation by Dr. Elisabet González, a researcher from the Nanomol-bio group and scientific coordinator of the Nano4Rare project. She introduced the audience to the project’s objectives, challenges, and vision for improving the treatment options available to people living with Fabry disease. Her talk set the tone for a day focused on innovation, collaboration, and patient-centered research.
The event brought together key stakeholders in the rare disease community, including clinicians such as Dr. Guillem Pintos and Dr. Maria Camprodon from Vall d’Hebron Hospital, who shared their insights on Fabry diagnosis and treatment from a clinical perspective. Renowned researcher Dr. Sílvia Muro, from the Institute for Bioengineering of Catalonia (IBEC), also delivered a keynote lecture on drug delivery strategies, offering a broader context on the state of the art in the field.
In addition to the scientific talks, the program featured a roundtable with patient associations, where representatives from Spanish Fabry and other rare disease organizations shared their experiences and outlined their needs and expectations regarding new therapies. This open dialogue between patients and scientists reinforced the importance of mutual understanding and co-design in biomedical research.
Participants also had the opportunity to visit the facilities of Nanomol Technologies, a spin-off company from the Nanomol-bio research group, where some of the innovative technologies used in the Nano4Rare project are being developed. This visit provided patients and guests with a closer look at how research is translated into potential therapies, and how advanced nanotechnologies are tailored to address complex diseases like Fabry.
By bridging the gap between science and the patient community, Fabry Connectionsexemplified the commitment of the Nano4Rare consortium—and especially the Nanomol-bio team—to making research more inclusive, responsive, and impactful. The event not only strengthened collaborations among the various stakeholders but also highlighted how technology transfer and nanomedicine innovation can contribute meaningfully to the future of rare disease treatment.
