Rare Disease Cohorts Funding

CLOSED: This funding call aims to enhance and develop cohort research to accelerate the discovery of mechanisms and new treatments in rare disease cohorts.

Internal GOSH and ICH applicants only.

Previous awards

In February 2018 we announced our first funding call and are pleased to announce the successful applicants. After careful consideration and external peer review, the panel selected to fund two projects:

Principal Investigator Project Title Award
Dr Simon Eaton Defining mitochondrial dysfunction in dermatomyositis: towards new treatment targets £78,343
Professor Shamima Rahman Mito-omics approach to investigate a paediatric cohort of multiple ultra-rare mitochondrial disorders £104,937

In July 2019 we announced our second funding call and are pleased to announce the successful applicants. After careful consideration and external peer review, the panel selected to fund three projects:

Principal Investigator Project Title Award
Professor Claire Booth Developing a gene editing therapy for XIAP deficiency £41,450
Professor Pia Hardelid Establishing a birth cohort for population-based follow-up of children with rare diseases £94,858
Professor Veronica Kinsler Genetic discovery in ILVEN leads to patient treatment £48,500

Call information

All projects will be considered, but priority will be given to those which both most closely align with the RD Theme’s strategy*, and which could clearly lead to patient benefit within the NHS, or which are most likely to lead to either external funding being leveraged. Co applicants from different disciplines are encouraged and collaborative applications from two different fields where a more senior applicant can provide mentorship to the more junior applicant will be given preference. Please note that to open the grant funding, successful applicants will need to show ethical approval for their project OR as a minimum, that submission to ethics has been done. The funding for this call will not be available until 01 April 2020 and your awarded money will be split across the two years, Y1's allocation must be spent by March 20th 2021 and cannot be rolled over. Y2's allocation will become available from 01 April 2021 and must be spent by March 20 2022. 

Please note the RDC theme strategy can be found at this link. In the interest of continued PPIE engagement, applicants should take extra care with their lay summaries (no technical terms, in plain English please). Applications that do not adhere to this may not be considered. 

Application process 

To apply please review and complete the application form (as well as register with R&D) obtaining the required signatures, and submit your CV and application form to BRC@gosh.nhs.uk by 27 September 2019.

R&D Registration_Form (Jul 19) (147.45 KB)

NIHR GOSH BRC RDC Theme Funding Call 2019 Application Form (194.41 KB)

Important dates

  • 19 July - application process opens
  • 6 September - deadline for registering application with R&D
  • 27 September - application deadline
  • Mid-November - panel review applications 
  • Late November - applicants informed of decision
  • 1 April - funding becomes available for successful applicants


If you intend to apply to this call, please read the RD Theme’s strategy on the GOSH-BRC website to ensure your application clearly aligns with the core theme. No animal research is supported. For any queries please contact Dauda Bappa.