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Our Research

Our five research goals are designed to drive improvement across all aspects of the Centre's work.

Our research goals

  1. We consolidate and enhance the multidisciplinary teams necessary for world-leading discovery research in rare disease
  2. We propose and employ innovative methodologies and resources to propel translational research from the lab to the clinic
  3. We continue to lead in policy generation and implementation. We, therefore, enhance research and impact on all aspects of health and wellbeing. We improve patient quality of life and bring stronger societal benefits
  4. We sustain and foster collaborations with patient organisations, policymakers, companies and regulators. We shape and seize new funding opportunities at national, European, and global levels
  5. We optimise teaching, education, and outreach around rare disease at Newcastle. We foster a pipeline of researchers and healthcare professionals to drive our future activities in this space

We believe a 'platform' approach to research is highly beneficial in rare disease. There's much to gain from collaborative research into common pathways (for instance signalling pathways) and downstream effects of disease.

Dr Neil Rajan

Patient-centred research

We know that patient participation in research design and management leads to better outcomes.

The rare disease patient community has a wealth of knowledge, which comes from lived experience. It can play a crucial role in shaping research. We define this broadly to include:

  • clinical interventions
  • drug development
  • lab/computer-based research
  • holistic care
  • policy

'We foster a dynamic dialogue between researchers and patients​ to encourage trust and confidence around research. In this way, we can be certain the research we're working on is relevant and needed by patients. And that is it carried out with integrity.'

Dr Pauline McCormack

See 'For Patients' for more details.