NIHR to deliver progress for rare disease patients with new investment

Following on from the Government’s publication of its new Rare Diseases Action Plan yesterday, the National Institute for Heath and Care Research (NIHR) is set to launch numerous new initiatives in the field that it says will drive progress for patients.

NIHR is rolling out funding to the tune of almost £800m for its Biomedical Research Centres (BRC) which will include support for research into a wide range of rare conditions like immunodeficiencies and haemophilia. It is also looking to develop a new Rare Disease Research Platform with a joint £12m funding call with the Medical Research Council.

Commenting on the launch of 2023’s Rare Diseases Action Plan, NIHR’s Chief Executive, Professor Lucy Chappell, explained: "The impacts of rare diseases on individuals and their families are wide-ranging. Our research plays an important underpinning role in furthering the understanding, diagnosis, treatment and care for people living with rare diseases.

“The action plan highlights significant advances in rare disease research supported through our funding. There is always more to do and our commitments will be supported by the recently announced funding for ground-breaking research into the NIHR Biomedical Research Centres and the Medical Research Council (MRC)-NIHR UK Rare Disease Research Platform.

“This research will continue to drive progress and foster collaboration to translate scientific breakthroughs into clinical advances.”

The £790m BRC investment will supplement research that has already supported:

  • The development of a new gene therapy that treats an immunodeficiency called ADA-SCID at Great Ormond Street;
  • The launch of the UK part of a global gene therapy trial investigating Duchenne muscular dystrophy in Newcastle;
  • The pioneering of new gene therapies that enable those with rare primary immunodeficiency disorders to live longer and better lives at University College London Hospitals;
  • The initialisation of a project that harnessed the power of genome sequencing to deliver timely diagnoses of rare conditions for severely ill babies and children in Cambridge.

More information on NIHR’s work researching rare diseases is available here.

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