A coordinated international initiative, TIRCON has set-up a framework for improvements in clinical practice and research.
As tangible achievements, TIRCON has successfully:
- Set up dedicated scientific research infrastructure for NBIA
- Conducted multi-center clinical trials and a biomarker study
- Established ethics approvals
- Generated the pre-clincal framework for future causative therapies
- Made scientific knowledge accessible to the NBIA community
TIRCON’s main outcome comprises:
- Structural impact: Through the cooperative effort of the major medical NBIA centers in Europe and the United States, NBIA patient advocacy groups and the biotech industry, TIRCON brought together patients and patient data, scientific and clinical expertise, and the participation of the biotech industry for the first time in a structured, well-networked form.
- Scientific impact: Structuring research in the field of NBIA at an international level ensures sufficient patient cohorts for statistical evidence and for the generation of solid scientific results in order to create new knowledge on NBIA that can be transferred nto more appropriate drug therapy strategies through a strong cooperation with industry.
- Economic impact: SME participation in TIRCON increases innovation and competitiveness of European health-related industries.
- Health and social impact: In the long run, TIRCON contributes to an improvement of the therapeutic options for a progressive, disabling, nd often life-threatening disease and leads to a distinctive improvement of the infrastructure for NBIA patients.