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SYMPHONY consortium: Orchestrating personalized treatment for patients with bleeding disorders
Marjon H Cnossen1, Iris van Moort2, Simone H Reitsma1
1Department of Pediatric Hematology and Oncology, Erasmus University Medical Center, Rotterdam, The Netherlands.
The SYMPHONY consortium aims to personalize treatment for inborn bleeding disorders by studying variations in bleeding. This research will improve diagnosis and treatment strategies for patients.
Area of Science:
- Hematology
- Genetics
- Personalized Medicine
Background:
- Increasing treatment options and costs for inborn bleeding disorders present challenges.
- An integrated, interdisciplinary national research program has been established to address these issues.
Purpose of the Study:
- To orchestrate personalized treatment for patients with inborn bleeding disorders.
- To unravel mechanisms behind inter-individual variations in bleeding phenotype.
Main Methods:
- Investigating both diagnosed and undiagnosed patients with inborn bleeding disorders.
- Research categorized under diagnosis, treatment, and fundamental research themes.
- Collaborations between patients and researchers from diverse expertise areas.
Main Results:
- Research questions encompass diagnosis, treatment, and fundamental research.
- Work packages address specific domains within these themes.
- Collaborations foster intellectual property and enhance research impact.
Conclusions:
- SYMPHONY will research all aspects of care and treatment individualization for inborn bleeding disorders.
- Focus on diagnostic innovations, molecular genetics, and cellular models of hemostasis.
- Expected health care innovations with long-term clinical and societal impact.
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