Neurodegeneration with Brain Iron Accumulation (
NBIA) is a group of very rare and mostly hereditary neurological movement disorders characterized by high iron levels in the basal ganglia and a progressive degeneration of the nervous system. Currently, there is no cure or proven therapy for any of the NBIA forms. Given the lack of treatment options, the international consortium TIRCON (Treat–Iron–Related–Childhood–Onset–Neurodegeneration), funded by the EU under FP7, is dedicated to the investigation of NBIA. 13 partners from eight countries are conducting a clinical therapy study and set up a patient registry/biobank in order to improve the infrastructure for NBIA research and care. We present the latest development, goals, recent and upcoming activities of the NBIA lay advocacy, thus demonstrating the rising impact of the growing NBIA Alliance and its member groups in interrelationship to the TIRCON consortium and to further NBIA research groups.
Methods
The lay advocacy groups, NBIA Disorders Association (NBIADA, USA) and Hoffnungsbaum e.V. (HoBa, Germany), are full partners in TIRCON. Based upon their experience in such collaborative projects, NBIADA and HoBa have been promoting the creation of further patient groups worldwide. The NBIA Alliance, launched under the patronage of TIRCON in 2012, is the only international umbrella association for NBIA lay advocacy groups. Currently, eight NBIA patient organizations are involved in the Alliance. The groups share the common goals of helping families cope with NBIA disorders, promoting improved health care in NBIA, and supporting research to find more effective treatments for these disorders.
Results
Current efforts include developing a strategic plan to support these common goals and expand the Alliance to other countries.
Conclusion
We will underline the mutual empowerment of the NBIA patients' movement and the NBIA research and care community.
