Nanomedicine and Rare Diseases: Advances and Collaboration at the Nano Rare Diseases Day 2025

Experts in nanomedicine from various fields gathered on March 7th, to present the latest advancements in the diagnosis and treatment of rare diseases. The Nano Rare Diseases Day, now in its sixth edition, is held within the framework of Rare Disease Day and is co-organized by NANOMED Spain and the Sant Joan de Déu Research Institute (IRSJD).

What Are Pediatric Rare Diseases?

Pediatric rare diseases are conditions that affect a small number of children and adolescents compared to the general population. In Europe, a disease is considered rare if it affects fewer than 5 people per 10,000 inhabitants. It is estimated that there are between 5,000 and 7,000 rare diseases, with around 4,000 still lacking treatment or a cure.

Nano Rare Diseases Day

The Nano Rare Diseases Day is an annual event held in the context of Rare Disease Day, aiming to showcase the latest advances in nanomedicine applied to these conditions. The event brings together researchers, clinicians, industry representatives, healthcare authorities, and patients to exchange knowledge and promote collaboration.

This year, in its sixth edition, the event welcomed over 50 participants, offering talks focused on the latest breakthroughs in nanomedicine applied to rare diseases, highlighting its potential for early diagnosis and the development of new therapies.

Multidisciplinary Collaboration Between Research Centers

The event was opened by Joan Comella, director of the Sant Joan de Déu Research Institute (IRSJD), and Josep Samitier, scientific coordinator of NANOMED Spain and director of the IBEC. Both emphasized the crucial role of interdisciplinary collaboration in addressing the challenges posed by these diseases and ensuring that scientific advances translate into real benefits for society.

Samitier stressed that "to bring research to patients, close collaboration between researchers, hospitals, and companies is essential. In this regard, one of the main goals of the Spanish Platform for Nanomedicine is to foster public-private collaboration initiatives, both nationally and internationally." Comella highlighted the IRSJD's collaborations and networks, aimed at accelerating research on pediatric rare diseases. He also emphasized the need for a specific approach to rare diseases in children, as they are often linked to developmental issues, whereas in adults, they tend to have a genetic origin.

The Essential Role of Patient Associations

Another key moment of the event was the discussion on the vital role of patient associations in research. Jordi Cruz, from the SJD Barcelona Children’s Hospital, and Eduard Goñalons, from the Fundación Noelia, shared their efforts to support patients and families affected by collagen VI-related muscular dystrophy (DMC-Col6).

The Rare Disease Day celebration and events like Nano Rare Diseases Day are essential for raising awareness about these conditions and promoting research and the development of new treatments. In this context, nanomedicine stands out as a key tool to tackle the challenges posed by rare diseases and improve both their diagnosis and treatment.

These types of gatherings are crucial to fostering research and innovation, as well as giving a voice to patients and their needs.