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News from the BRC

  • Professor Wedderburn First large-scale genetic association study of drug response in childhood arthritis A study led by Professor Wedderburn in collaboration with health research centres in Manchester, Birmingham, the Netherlands and Czech Republic has discovered a potential explanation for the differences in drug treatment response. Three novel genes have been identified as being likely to be associated with response to the drug methotrexate.
  • Sophia Varadkar Two patients recruited at GOSH to natural history study of ultra rare metabolic disorderMolybdenum cofactor deficiency (MoCD) type A is an ultra rare metabolic disorder that untreated results in neurological damage and death. 40 centres worldwide are participating in a natural history of MoCD that will inform future therapeutic trials. Within a few weeks of study opening, a team led by Dr Sophia Varadkar has achieved recruitment of two patients to the trial.
  • Bobby Gaspar and Adrian Thrasher in ICH lobby Development of a stem cell gene therapy trial in children with Sanfilippo diseaseThe BRC funded Gene and Cell Therapy Facility and Great Ormond Street Hospital Children's Charity are supporting the development of a stem cell gene therapy trial for in children with Sanfilippo disease. The benefit to patients from this technology is likely to be a lifelong therapy, which could help to improve brain disease.

Films about the BRC

Introduction to the work done at the BRC
View on YouTube.
Generation R: Young people in research
View on YouTube.
Homepage banner for BRC Real Story

Useful links

Find out more about the NIHR GOSH BRC Open Day and London Science Festival on Saturday, 15 November at UCL Institute of Child Health


To read a report on achievements by our BRC in the last two years, please take a look at our BRC brochure.

Contact us

GOSH Biomedical
Research Centre

UCL Institute of Child Health
R&D Office
30 Guilford Street 
London WC1N 1EH

E-mail: GOSH BRC Coordinator: Danielle.Wagner@gosh.nhs.uk

Phone: 0207 905 2844