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).
Earlier this month patients and families at GOSH (Great Ormond Street Hospital) were given the chance to get hands-on with science at our Research Activity Trial and with special lessons in the hospital school.