A new collaboration aiming to develop advanced technologies for manufacturing ex vivo gene therapy products has been established between researchers at UCL Great Ormond Street Institute of Child Health (ICH) and Plasticell, a developer of stem cell technologies.
Four collaborative research trials, which all included GOSH patients treated at the National Institute for Health Research (NIHR) GOSH Clinical Research Facility (CRF), have been recently published in the prestigious New England Journal of Medicine.
Dr Gabriel Galea has been awarded a Wellcome Trust Clinical Research Career Development Fellowship to continue important research into the molecules, genes and physical forces that control the formation of the neural tube in the developing embryo.
The number of people taking part in research trials at Great Ormond Street Hospital (GOSH) has increased by 48 per cent, according to figures released this week by the National Institute for Health Research (NIHR).
Professors Graham Davies and Adrian Thrasher have been awarded funding from Great Ormond Street Hospital (GOSH) Charity to further develop a cutting-edge new treatment for children with the rare immunodeficiency condition complete DiGeorge syndrome (cDGS).
A new, targeted drug has been shown to significantly improve growth and control of rickets in children with X-linked hypophosphataemic rickets (XLH), a serious condition affecting about 300 children in the UK.
XLH is caused by genetic mutation that leads to excess activity of fibroblast...
Giving the drug sodium thiosulphate after chemotherapy reduces hearing loss in children treated for liver cancer, according to findings published in the New England Journal of Medicine today (Wednesday).
Cannabidiol, a drug derived from cannabis but with the psychoactive ingredients removed, has been shown to reduce seizures by around 40 % in children with a rare form of drug resistant epilepsy known as Lennox-Gastaut syndrome (LGS).