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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

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