Rare Diseases Foundation
The Dutch Rare Diseases Foundation (Zeldzame Ziekten Fonds) helps people with rare diseases. They do this by raising money for scientific research, raising awareness of rare diseases and facilitating peer-to-peer contact. We have a long-term partnership with the Dutch Rare Diseases Foundation. Through our contribution, we hope to raise awareness and enable the development of proper treatment for one of the many rare diseases.
Studies we have contributed to include:
Innovative research into severe bone marrow failure in children
In January 2024, research started on a rare disease that impedes the functioning of bone marrow in children. About 10 children in the Netherlands experience this severe form of bone marrow failure every year, and researchers often do not know why. Children with this life-threatening condition are usually treated with a stem cell transplant, which is an intensive treatment with many side effects.
The most appropriate treatment
Some children could possibly be treated with immune-suppressing drugs instead of a stem cell transplant. To investigate this, researchers at the Willem-Alexander Children's Hospital (WAKZ) of Leiden University Medical Center, led by Alexander Mohseny, have been collecting blood and bone marrow samples from children with bone marrow failure for the past few years.
The aim of this study is to identify the cause of bone marrow failure and then determine the most appropriate treatment for each patient. This may lead to treatments with fewer side effects.
By supporting the Dutch Rare Diseases Foundation’s 'Bone Marrow Failure in Children' research project, we hope researchers can make progress over the next 4 years to contribute to a healthier future for children with this rare condition.
Research on stem cell transplants in children with a rare disease
In June 2022, Inge Wessels visited Leiden University Medical Center with Mirre de Noo. There, they were given a presentation by Professor Arjan Lankaster on stem cell transplants in children with a rare disease. It was an impressive visit, showing how important and life-saving stem cell transplants are for these sick children.
‘We’re incredibly impressed by the drive of the team of healthcare professionals and researchers who have been working for years to improve not only the science, but also the quality of life of sick children and their parents/families.’
- Mirre de Noo
At Reggeborgh Foundation, we believe that health is the basis for everyone's happiness. That is why we invest in a variety of ways to ensure a healthy future for everyone in the Netherlands. Want to know more about this research? Read more on the Dutch Rare Diseases Foundation’s website or follow developments on social media.