Carte Blanche: World Rare Disease Day2026
From pregnancy to adulthood: paediatrics as the backbone of life pathways
On World Rare Disease Day, it is important to recall a fundamental reality: rare diseases are not merely diagnoses; they are life pathways. They are long and complex journeys, marked by uncertainty, the need for coordinated care, and continuous support — sometimes beginning before birth and extending into adulthood.
In Belgium, the Children’s Hospital (HUDERF) holds a unique position in this regard. It is the only exclusively paediatric hospital in the country. Integrated within the Brussels University Hospital, it benefits from a university-based and interinstitutional framework that enables care pathways for children with rare diseases to be organised in full continuity.
This distinctiveness goes beyond advanced paediatric expertise. It lies in a rare capacity: accompanying children and their families from the first days of life — or even several months before birth — through to the transition to adult medicine.
Through close collaboration with the Erasme Hospital, certain situations can be identified, discussed and prepared during pregnancy. The prenatal period is decisive. It allows needs to be anticipated, teams to prepare, parents to be informed and supported, and a care pathway to be structured at a very early stage. For many rare diseases, this already represents a first essential step.
The first days of life — and more broadly the first 1,000 days — constitute another key phase. Now widely recognised as fundamental for development, health and future trajectories, this period lies at the heart of the organisation of HUB – Brussels University Hospital. The neonatology teams at Erasme Hospital and the Children’s Hospital (HUDERF) ensure continuity between birth, specialised neonatal care, early follow-up and entry into specialised paediatric pathways. For many rare conditions, major decisions are taken at this stage, both medically and in terms of family support.
Throughout childhood and adolescence, the Children’s Hospital (HUDERF) provides specifically paediatric care based on a multidisciplinary approach bringing together specialised paediatricians, geneticists, neurologists, specialists in metabolic diseases, surgeons — notably in neurosurgery and interventional neurology — as well as psychologists, social workers and paramedical and educational teams.
Rare diseases are never confined to a single organ or symptom. They affect development, schooling, social participation, mental health and future autonomy. University paediatrics allows these dimensions to be integrated into a comprehensive approach centred on the child, their family and their environment.
These pathways do not end at the age of majority. For many young people living with a rare disease, the transition to adult medicine is a critical stage, often associated with disruption and fragmentation of care. The integration of the Children’s Hospital (HUDERF) within HUB – Brussels University Hospital, and its structural links with Erasme Hospital, make it possible to develop coordinated transition processes, both medically and psychosocially.
This is not a simple administrative transfer of a file. It is a gradual, interdisciplinary and interinstitutional process designed to ensure genuine continuity, taking into account the full spectrum of needs: medical, cognitive, psychological, social and familial.
Some children may therefore be followed:
- from pregnancy, before birth
- during the first 1,000 days
- throughout childhood and adolescence
- and supported in their transition to adult medicine
within a single integrated university framework linking the Children’s Hospital (HUDERF), Erasme Hospital and the Jules Bordet Institute.
Such continuity is rare. It is invaluable. It represents a major strength for quality of care, research and innovation — but above all for families, who find within this framework a stable point of reference in pathways often marked by complexity and uncertainty.
At a time when national rare disease plans, European reference networks, precision medicine and integrated care pathways are being developed, the experience of the Children’s Hospital (HUDERF) and HUB – Brussels University Hospital illustrates concretely what a life-course approach means. It recognises that rare diseases are not only rare; they are demanding — for patients, families and all professionals involved in care.
On this World Rare Disease Day, it is essential to emphasise that paediatrics is not simply one chapter in the pathway: it is often its backbone.
In Belgium, having a university hospital exclusively dedicated to paediatrics, integrated within a major university hospital and capable of accompanying children from the prenatal stage through to adulthood, is a major asset for the quality, equity and humanity of care.
Béatrice Gulbis, Professor and biologist doctor at the H.U.B
Franck Devaux, Ethicist and Coordinator of the Rare Diseases Unit of the H.U.B